miércoles, 30 de julio de 2014

Press Announcements > FDA expands approved use of Imbruvica for chronic lymphocytic leukemia

Press Announcements > FDA expands approved use of Imbruvica for chronic lymphocytic leukemia



FDA expands approved use of Imbruvica for chronic lymphocytic leukemia

New clinical data supports traditional approval for CLL

For Immediate Release

July 28, 2014

Release

The U.S. Food and Drug Administration today expanded the approved use of Imbruvica (ibrutinib) to treat patients with chronic lymphocytic leukemia (CLL) who carry a deletion in chromosome 17 (17p deletion), which is associated with poor responses to standard treatment for CLL. Imbruvica received a breakthrough therapy designation for this use.
The FDA is also approving new labeling to reflect that Imbruvica’s clinical benefit in treating CLL has been verified. In February 2014, Imbruvica received accelerated approval to treat CLL based on its effect on overall response rate. New clinical trial results examining progression-free survival and overall survival have confirmed the drug’s clinical benefit.
A type of non-Hodgkin lymphoma, CLL is a rare blood and bone marrow disease that usually gets worse slowly over time, causing a gradual increase in white blood cells called B lymphocytes, or B cells. The National Cancer Institute estimates that 15,720 Americans will be diagnosed and 4,600 will die from CLL in 2014. Imbruvica works by blocking the enzyme that allows cancer cells to grow and divide.
“We continue to see advances in the availability of therapies to treat chronic lymphocytic leukemia, especially for difficult-to-treat patient populations,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Imbruvica is the fourth drug approved to treat CLL that received a breakthrough therapy designation, reflecting the promise of the breakthrough therapy designation program and demonstrating the FDA’s commitment to working cooperatively with companies to expedite the development, review and approval of these important new drugs.” 
The other three drugs approved to treat CLL that received breakthrough designations are Gazyva (obinutuzumab) in November 2013, Arzerra (ofatumumab) in April 2014 and Zydelig (idelalisib) in July 2014. Imbruvica’s application for accelerated approval to treat CLL did not receive breakthrough therapy designation.
Today’s approval actions for Imbruvica are based on a clinical study of 391 previously treated participants, 127 of whom had CLL with 17p deletion. Participants were randomly assigned to receive Imbruvica or Arzerra until disease progression or side effects became intolerable.
The trial was stopped early for efficacy after a pre-planned interim analysis showed Imbruvica-treated participants experienced a 78 percent reduction in risk of disease progression or death (progression-free survival). Results also showed a 57 percent reduction in risk of death (overall survival) in participants treated with Imbruvica. Of the 127 participants who had CLL with 17p deletion, those treated with Imbruvica experienced a 75 percent reduction in risk of disease progression or death.
The most common side effects associated with Imbruvica observed in the clinical study include low levels of platelets in the blood (thrombocytopenia), a decrease in infection-fighting white blood cells called neutrophils (neutropenia), diarrhea, low red blood cells (anemia), fatigue, pain in the muscles and bones (musculoskeletal pain), upper respiratory tract infection, rash, nausea and fever (pyrexia).
Imbruvica’s new use is being approved more than two months ahead of the product’s prescription drug user fee goal date of Oct. 7, 2014, the date the FDA was scheduled to complete review of the drug application. The FDA reviewed Imbruvica’s application for this new use under the agency’s priority review program, which provides for an expedited review of drugs that are intended to treat a serious disease or condition and, if approved, would offer significant improvement compared to marketed products.
Imbruvica also received accelerated approval in November 2013 for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy. Clinical studies to verify and describe Imbruvica’s clinical benefit in mantle cell lymphoma are ongoing. 
Imbruvica is co-marketed by Pharmacyclics, based in Sunnyvale, Calif., and Janssen Biotech, based in Horsham, Penn.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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Press Announcements > FDA Commissioner Margaret A. Hamburg’s Statement on the Surgeon General’s Call to Action to Prevent Skin Cancer

Press Announcements > FDA Commissioner Margaret A. Hamburg’s Statement on the Surgeon General’s Call to Action to Prevent Skin Cancer



FDA Commissioner Margaret A. Hamburg’s Statement on the Surgeon General’s Call to Action to Prevent Skin Cancer

For Immediate Release

July 29, 2014

Statement

Each year, thousands of Americans are diagnosed with some form of skin cancer. The Surgeon General’s Call to Action to prevent skin cancer is important especially during these hot summer months when many of us spend extra time in the sun.
Over the last few years, the FDA has taken a number of important steps designed to help consumers better understand the harmful effects of exposure from the sun and from indoor tanning.
Today, consumers going to a beach or pool can rely on more accurate information on the labels of all sunscreen products on the market. In 2011, the FDA made changes that help consumers buy and use sunscreen. Consumers now see accurate labels that may include “Broad Spectrum” claims and water resistance claims (how long a sunscreen remains effective while swimming or sweating).
Earlier this year, the agency changed its risk classification for sunlamp products (e.g., tanning beds, tanning booths), imposing increased regulatory controls on these products. The products now must be labeled with boxed warnings stating that they should not be used on anyone younger than 18 years, and specific contraindications and warnings must be included in certain promotional materials for these products.
These were important actions and steps that the FDA hopes will help address the harmful effects of the sun and UV radiation exposure.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Safety Alerts & Advisories > FDA Consumer Advice on Powdered Pure Caffeine

Safety Alerts & Advisories > FDA Consumer Advice on Powdered Pure Caffeine



FDA Consumer Advice on Powdered Pure Caffeine

(In English and Spanish / En inglés y español)

Key Message

The FDA is warning about powdered pure caffeine being marketed directly to consumers, and recommends avoiding these products.  In particular, FDA is concerned about powdered pure caffeine sold in bulk bags over the internet.
The FDA is aware of at least one death of a teenager who used these products.
These products are essentially 100 percent caffeine. A single teaspoon of pure caffeine is roughly equivalent to the amount in 25 cups of coffee.
Pure caffeine is a powerful stimulant and very small amounts may cause accidental overdose. Parents should be aware that these products may be attractive to young people.
Symptoms of caffeine overdose can include rapid or dangerously erratic heartbeat, seizures and death. Vomiting, diarrhea, stupor and disorientation are also symptoms of caffeine toxicity. These symptoms are likely to be much more severe than those resulting from drinking too much coffee, tea or other caffeinated beverages.

Who should know

All consumers seeking caffeinated products should be aware of the potentially high potency of these powdered pure caffeine products. Parents should recognize that teenagers and young adults may be drawn to these products for their perceived benefits.

What to do

  • The FDA advises consumers to avoid powdered pure caffeine.
  • It is nearly impossible to accurately measure powdered pure caffeine with common kitchen measuring tools and you can easily consume a lethal amount.
  • If you believe that you are having an adverse event related to caffeine, stop using it and seek immediate medical care or advice.
  • The FDA wants to know about adverse events associated with powdered pure caffeine and other highly caffeinated products. You or your health care provider can help by reporting these adverse events to FDA in the following ways:
  • By phone at 240-402-2405
  • By email at CAERS@cfsan.fda.gov

Why this advice is important

Pure caffeine products are potentially dangerous, and serious adverse events can result, including death. People with pre-existing heart conditions should not use them.

Consumer Updates > Faster, Easier Cures for Hepatitis C

Consumer Updates > Faster, Easier Cures for Hepatitis C



Faster, Easier Cures for Hepatitis C

Faster, Easier Cures for Hepatitis C (350x240)


On this page:
Transformative advances in drug treatments approved by the Food and Drug Administration are giving the 3.2 million Americans with chronic hepatitis C a chance for a longer, healthier life without the virus. That’s welcome news for baby boomers—who make up three of four adults with the hepatitis C virus—and millions of other Americans, many of whom don’t yet know they are infected and carriers.
Hepatitis C can be cured, and today’s drug therapies are very effective and easier for patients to take, says Jeffrey S. Murray, M.D., the deputy director of the Division of Antiviral Products in FDA’s Center for Drug Evaluation and Research. Murray is an internist who specializes in infectious diseases.

A Preventable and Curable Disease

Hepatitis (inflammation of the liver) refers to a group of viral infections that affect the liver. The most common types are hepatitis A, hepatitis B and hepatitis C. Each is caused by a different virus.
Hepatitis C is the most common chronic blood-borne infection in the United States. There is no vaccine for this disease, but hepatitis C can be prevented by avoiding behaviors that can spread the virus—including sharing needles, syringes or other equipment to inject drugs.
A diagnosis of hepatitis C no longer means months and months of painful drug injections, which for decades were the only option. Science is making strides in therapies, giving patients new alternatives.
“Interferon-based injections often make patients feel ill and give them flulike symptoms,” Murray says. The treatment by interferon also lasts six months to a year, and cures only 40% to 50% of hepatitis C patients.
“Patients with very advanced liver disease couldn’t take the traditional treatment because often those injections could make them worse,” he adds. “Now, patients can treat their hepatitis C with only pills– drug combinations that are faster and have a higher cure rate.”
Today’s pills have double the viral cure rates—90% to 100%—in just in 12 weeks’ time. Reducing the treatment from a year to three months is a huge advantage for people with hepatitis C, especially because it’s easier to swallow a pill than to get an injection, Murray says.
The new regimens include Sovaldi (sofosbuvir), which is the first drug approved to treat certain types of hepatitis C infection without the need to co-administer interferon. In recent years, FDA has also approved three protease inhibitors—Olysio (simeprevir), Victrelis (boceprevir) and Incivek (telaprevir)—to treat chronic hepatitis C virus infection. Olysio is a protease inhibitor that blocks a specific protein the hepatitis C virus needs to replicate. The drug is a component of a combination antiviral treatment regimen.
FDA provides information through a Hepatitis e-mails list, along with notices of upcoming public events, such as advisory committee meetings, and opportunities to comment on policies and issues that affect people with hepatitis B or C.

Baby Boomers and Hepatitis C

For most people, hepatitis is a silent disease until it causes substantial damage to the liver. That process may take several years, and can lead to liver failure, liver transplantation and liver cancer.
“Hepatitis C is a bit like smoking, the longer you’ve had it, the higher your risk of developing complications—in this case, liver cancer and end-stage liver disease. It’s a progressive disease that takes years, even decades, before the patient develops cirrhosis or cancer,” Murray says. “The good news is that when you cure hepatitis C, you also lower its risks, though you don’t completely erase the years of damage to your liver.”
Once infected with the hepatitis C virus, nearly 8 in 10 untreated people remain infected for life, according to theCenters for Disease Control and Prevention (CDC). Three in four patients with chronic hepatitis C are baby boomers (people born from 1945 to 1965), and many became infected before the virus was identified and the blood supply was tested for the disease. That’s why it’s important for baby boomers—there are 76.4 million of them, according to the U.S. Census Bureau—to take a simple blood test for hepatitis C.
“When it comes to hepatitis C, the outlook for the future is better, but the past is catching up with us—especially if you are a baby boomer,” Murray says. “Still, this is a fortuitous time because better hepatitis C treatments are becoming available just as the patient population at risk of long-term complications is about to peak. There are treatments for chronic hepatitis and many reasons to get tested now more than ever because of the availability of safe and effective therapies.”
This article appears on FDA’s Consumer Updates page, which features the latest on all FDA-regulated products.
July 28, 2014

blog.aids.gov − Join World Hepatitis Day at the White House – July 30: A Call to Action for Public Health Leadership

blog.aids.gov − Join World Hepatitis Day at the White House – July 30: A Call to Action for Public Health Leadership



AIDS.gov Blog Update

AIDS.gov Blog for U.S. Dept. of Health & Human Services.
This information has recently been updated, and is now available.
07/30/2014 09:21 AM EDT

Every year, on July 28th, we commemorate World Hepatitis Day and elevate the public health response to a disease that impacts more than 400 million people globally. It has been an honor to serve as Assistant Secretary of Health (ASH) at the US Department of Health and Human Services for the past five years. This...

Latest marketing authorisations and orphan drug designations

Latest marketing authorisations and orphan drug designations

Marketing authorisations and medicinal products designations

Detailed information on European orphan medicinal products designation applications is available on theEMA website. A full list of designated and authorised orphan medicinal products in Europe available at:ec.europa.eu.

Orphan drug regulatory processLearn more about the Orphan designation process in Europe




Recent marketing authorisations

  • Deltyba (delamanid)

Treatment of tuberculosis
Otsuka Novel Products GmbH
Germany
28 April 2014
What is Deltyba and what is it used for?
Deltyba is a tuberculosis medicine that contains the active substance delamanid. Tuberculosis is an infection caused by the bacterium Mycobacterium tuberculosis (M. tuberculosis). Deltyba is used in adults with tuberculosis that is affecting the lung and that is multi-drug resistant (resistant to at least isoniazid and rifampicin, two standard anti-tuberculosis medicines). It is used together with other standard medicines and when other combinations without this medicine cannot be used either because the disease is resistant to them or because of their side effects.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Deltyba was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 1 February 2008.
 
  • Para-aminosalicylic acid Lucane (para-aminosalicylic acid (PAS))

Treatment of tuberculosis
Lucane Pharma SA
France
07 April 2014
What is Para-aminosalicylic acid Lucane and what is it used for?
Para-aminosalicylic acid Lucane is a tuberculosis medicine that contains the active substance para-aminosalicylic acid (PAS). It is used in combination with other medicines to treat adults and children from 28 days of age who have multi-drug resistant tuberculosis when combinations without this medicine cannot be used, either because the disease is resistant to them or because of their side effects.
Multi-drug resistance is when the bacteria causing tuberculosis (Mycobacterium tuberculosis) are resistant to treatment with at least isoniazid and rifampicin, two standard tuberculosis medicines.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Para-aminosalicylic acid Lucane was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 December 2010.

  • Cholic acid FGK (cholic acid)

Treatment of inborn errors in primary bile acid synthesis
FGK Representative Service GmbH
Germany
04 April 2014
What is Cholic acid FGK and what is it used for?
Cholic acid FGK is a medicine that contains the active substance cholic acid. This is a ‘primary bile acid’, which is a main component of the bile (a fluid produced by the liver that helps to digest fats).
Cholic acid FGK is used for the life-long treatment of adults and children from one month of age who cannot produce enough primary bile acids such as cholic acid due to genetic abnormalities that result in a lack of liver enzymes. When these primary bile acids are lacking, the body produces abnormal bile acids instead, which can damage the liver potentially leading to life-threatening liver failure. The condition is known as ‘inborn errors in primary bile acid synthesis’.
Cholic acid FGK is authorised for use in those patients who lack one of the following liver enzymes: sterol 27-hydroxylase; 2-methylacyl-CoA racemase; or cholesterol 7α-hydroxylase.
Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Cholic acid FGK was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 October 2009.
 
  • Cometriq (cabozantinib)

Treatment of medullary thyroid carcinoma
TMC Pharma Services Ltd
United-Kingdom
21 March 2014
What is Cometriq and what is it used for?
Cometriq is a cancer medicine that contains the active substance cabozantinib. It is used to treat adults with medullary thyroid cancer, a type of cancer originating in the cells in the thyroid gland that produce the hormone calcitonin. Cometriq is used when the cancer cannot be removed by surgery and has progressed or spread to other parts of the body.
The benefits of Cometriq may be smaller for patients whose cancer does not have a mutation in a gene called the ‘re-arranged during transfection’ (RET) gene, and this should be taken into account when deciding whether to start treatment.
Because the number of patients with medullary thyroid cancer is low, the disease is considered ‘rare’, and Cometriq was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 February 2009.

  • Sirturo (bedaquiline)

Treatment of tuberculosis
Janssen-Cilag International N.V.
Belgium
5 March 2014
What is Sirturo and what is it used for?
Sirturo is a tuberculosis medicine that contains the active substance bedaquiline. Tuberculosis is an infection caused by the bacterium Mycobacterium tuberculosis. Sirturo is used in combination with other tuberculosis medicines in adults with tuberculosis that is affecting the lung and that is multi-drug resistant (resistant to at least isoniazid and rifampicin, two standard tuberculosis medicines). It is given when combinations without Sirturo cannot be used, either because the disease is resistant to them or because of their side effects.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Sirturo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 August 2005.

  • Adempas (riociguat)

Treatment of Pulmonary arterial hypertension (PAH), including treatment of chronic thromboembolic pulmonary hypertension (CTEPH)
Bayer Pharma AG
Germany
27 March 2014
What is Adempas and what is it used for?
Adempas is a medicine that contains the active substance riociguat. It is used to increase the ability to carry out physical activity in adults with the following forms of pulmonary hypertension (high blood pressure in the blood vessels of the lungs):
  • Chronic thromboembolic pulmonary hypertension (CTEPH, where the blood vessels of the lungs are blocked or narrowed with blood clots). Adempas is used to treat patients with CTEPH who cannot be operated on, or in whom CTEPH remains or returns after surgery.
  • Pulmonary arterial hypertension (PAH, where the walls of the blood vessels of the lungs are thickened and the vessels become narrowed). Adempas can be used on its own or in combination with other medicines for PAH called ‘endothelin receptor antagonists’.

Adempas is used in patients with functional class II to III CTEPH or PAH. The ‘class’ reflects the seriousness of the disease: ‘class II’ involves slight limitation of physical activity while ‘class III’ involves marked limitation of physical activity.
Because the number of patients with CTEPH or with PAH is low, the diseases are considered ‘rare’, and Adempas was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 December 2007.

  • Opsumit (macitentan)

Treatment of pulmonary arterial hypertension (PAH)
Actelion Registration Ltd
United-Kingdom
20 December 2013
What is Opsumit and what is it used for?
Opsumit is a medicine that contains the active substance macitentan. It is used for the long-term treatment of pulmonary arterial hypertension (PAH), a condition in which there is abnormally high blood pressure in the arteries of the lungs, causing symptoms such as breathlessness and fatigue.
Opsumit is used for adults whose PAH is classified as ‘WHO functional class II to class III’. The ‘class’ reflects the seriousness of the disease: patients with class II PAH have slight limitation of physical activity and those with class III disease have marked limitation of physical activity. Opsumit can be used alone or in combination with other PAH medicines; for further information, see the package leaflet.
Because the number of patients with PAH is low, the disease is considered ‘rare’, and Opsumit was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 September 2011.

 
  • Orphacol (cholic acid)

Treatment of inborn errors in primary bile acid synthesis
Laboratoire CTRS
France
12 September 2013
What is Orphacol and what is it used for?
Orphacol is a medicine containing cholic acid, a substance found in the bile which is used to digest fats.
It is used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. Orphacol is used in patients who do not have enough of two specific liver enzymes (3β-Hydroxy-Δ5-C27-steroid oxidoreductase or Δ4-3-Oxosteroid-5β-reductase). This makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure.
Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Orphacol was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 December 2002.

  • Defitelio (defibrotide)

Treatment of severe hepatic veno-occlusive disease (VOD)
Gentium S.p.A
Italy
18 October 2013
What is Defitelio and what is it used for?
Defitelio is a medicine containing the active substance defibrotide. It is used to treat severe veno-occlusive disease (VOD) in patients undergoing haematopoietic (blood) stem-cell transplantation. VOD is a condition in which the veins in the liver become blocked, leading to liver dysfunction. Defitelio is used in adults and in children from one month of age.
Because the number of patients with VOD is low, the disease is considered ‘rare’, and Defitelio was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 July 2004.
 
  • Imnovid (pomalidomide)
     

Treatment of multiple myeloma
Celgene Europe Ltd
United Kingdom
5 August 2013
What is Imnovid and what is it used for?
Imnovid is an anticancer medicine that contains the active substance pomalidomide. It is used in combination with dexamethasone (an anti-inflammatory medicine) to treat multiple myeloma (a cancer of the bone marrow). It is used in adults who have received at least two prior therapies, including both lenalidomide and bortezomib, and whose disease progressed after the last treatment.
Because the number of patients with multiple myeloma is low, the disease is considered ‘rare’, and Imnovid was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.
 
  • Procysbi (cysteamine (as mercaptamine bitartrate))
     

Treatment of nephropathic (kidney)cystinosis
Raptor Pharmaceuticals Europe
The Netherlands
06/09/2013
What is Procysbi and what is it used for?
Procysbi is a medicine that contains the active substance mercaptamine (also known as cysteamine). It is used in patients with nephropathic (kidney) cystinosis. Cystinosis is an inherited disease in which excess amounts of cystine, an amino acid naturally found in the body, build up within cells, especially in the kidneys and the eyes, damaging them.
Because the number of patients with cystinosis is low, the disease is considered ‘rare’, and Procysbi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 September 2010.
Procysbi is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Procysbi is available in a formulation that allows for a delayed release of the active substance in the body. The reference medicine for Procysbi is Cystagon.

  • Iclusig (ponatinib)
     

Treatment of chronic myeloid leukaemia (CML) ; acute lymphoblastic leukaemia (ALL)
Ariad Pharma Ltd
United-Kingdom
01/07/2013
What is Iclusig and what is it used for?
Iclusig is an anticancer medicine that contains the active substance ponatinib. It is used to treat adults with the following types of leukaemia (cancer of the white blood cells):
  • chronic myeloid leukaemia (CML) in its different stages known as chronic, accelerated and blast phases;
  • acute lymphoblastic leukaemia (ALL) in patients who are ‘Philadelphia-chromosome-positive’ (Ph+). Ph+ means that some of the patient’s genes have rearranged themselves to form a special chromosome called the Philadelphia chromosome that leads to the development of leukaemia. The Philadelphia chromosome is found in some ALL patients but is present in most patients with CML.
     
Iclusig is used in patients who cannot tolerate or do not respond to dasatinib or nilotinib (other anticancer medicines) and for whom subsequent treatment with imatinib is not considered appropriate. It is also used in patients who have a genetic mutation called ‘T315I mutation’ which makes them resistant to treatment with imatinib, dasatinib or nilotinib.
Because the numbers of patients with CML and ALL are low, the diseases are considered ‘rare’, and Iclusig was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 2 February 2010.

  • Revlimid (lenalidomide)
     

Extension of indication:
Treatment of myelodysplastic syndromes
Celgene Europe Ltd.
United Kingdom
25/04/2013
What is Revlimid?
Revlimid is a medicine that contains the active substance lenalidomide. It is available as capsules (2.5 mg, 5 mg, 7.5 mg, 10 mg, 15 mg and 25 mg).
What is Revlimid used for?
Revlimid is an anticancer medicine originally indicated to treat adults with multiple myeloma.
On 25 April 2013, the CHMP adopted the following new therapeutic indication for Revlimid: myelodysplastic syndromes.
Revlimid is indicated for the treatment of patients with transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.
 
  • Bosulif (bosutinib)
     

Treatment of adults with chronic myeloid leukaemia (CML)
Pfizer Limited
United-Kingdom
27/03/2013
Bosulif is an anticancer medicine that contains the active substance bosutinib. It is used to treat adults with chronic myeloid leukaemia (CML), a cancer of the white blood cells in which granulocytes (a type of white blood cell) start growing out of control. 
Bosulif is used in patients who are ‘Philadelphia-chromosome-positive’ (Ph+). This means that some of the patient’s genes have re-arranged themselves to form a special chromosome called the Philadelphia chromosome. Bosulif is used to treat three stages of CML called ‘chronic phase’, ‘accelerated phase’ and ‘blast phase’. It is only used when the CML has already been treated with one or more tyrosine kinase inhibitors (medicines for CML which work in a similar way to Bosulif) and when the tyrosine kinase inhibitors called imatinib, nilotinib and dasatinib are not considered appropriate treatment options. 
Because the number of patients with CML is low, the disease is considered ‘rare’, and Bosulif was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 August 2010.

  • NexoBrid (concentrate of proteolytic enzymes enriched in bromelain)
     

Treatment of partial deep dermal and full thickness burns
Teva Pharma GmbH
Germany
27/03/2013
What is NexoBrid?
NexoBrid is a medicine that contains the active substance ‘concentrate of proteolytic enzymes enriched in bromelain’. It is available as a powder and gel, which are mixed together to make a gel (2 g/22 g or 5 g/55 g).
What is NexoBrid used for?
NexoBrid is used in adults to remove eschar (dead tissue which is dried-out, thick, leathery and black) from deep partial thickness and full thickness burns of the skin caused by heat or fire. Deep partial thickness burns (sometimes called ‘second degree’ burns) extend into a deep region of an inner layer of the skin called the dermis, while full thickness burns (sometimes called ‘third degree’ burns) extend even deeper, through the whole dermis.
Because the number of patients with deep partial thickness and full thickness thermal burn wounds is low, the disease is considered ‘rare’, and NexoBrid was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 30 July 2002.
The medicine can only be obtained with a prescription.

  • Glybera (alipogene tiparvovec)
     

Treatment of lipoprotein lipase deficiency
uniQure biopharma B.V.
The Netherlands
25/10/2012
 
What is Glybera?
Glybera is a medicine that contains the active substance alipogene tiparvovec. It is available as a solution for injection.
Glybera is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.
What is Glybera used for?
Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.
Lipoprotein lipase deficiency is a rare disease in which patients have a defect in the gene for lipoprotein lipase, an enzyme responsible for breaking down fats. Patients with this disease need to be on a strict low-fat diet and are prone to recurring attacks of pancreatitis, which is a severe and life-threatening complication.
Glybera is only for patients whose disease has been confirmed by appropriate genetic testing and who have detectable levels of the lipoprotein lipase enzyme in their blood.
Because the number of patients with lipoprotein lipase deficiency is low, the disease is considered ‘rare’, and Glybera was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 March 2004.
The medicine can only be obtained with a prescription.
 
  • Adcetris ( brentuximab vedotin)
     

Treatment of Hodgkin lymphoma
Takeda Global Research and Development Centre (Europe) Ltd
United Kingdom
25/10/2012
What is Adcetris?
Adcetris is a medicine that contains the active substance brentuximab vedotin. It is available as a powder that is made up into a solution for infusion (drip into a vein).
What is Adcetris used for?
Adcetris is used to treat adults with Hodgkin lymphoma (HL, a type of cancer that originates from blood cells in the lymphatic system, a part of the immune system) when the tumour cells are CD30-positive (when they have a protein called CD30 on their surface). It is used:
when the cancer has come back or has not responded to an autologous stem cell transplant (a transplant of the patient's own blood-producing cells);
when the cancer has come back or has not responded to at least two previous therapies and when autologous stem cell transplant or multi-agent chemotherapy (a combination of anticancer medicines) are not treatment options.
Adcetris is also used to treat systemic anaplastic large cell lymphoma (sALCL, a CD30-positive cancer of white blood cells called T lymphocytes), when the cancer has come back or has not responded to other treatments.
Because the number of patients with HL and sALCL is low, the diseases are considered ‘rare’, and Adcetris was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 January 2009.
The medicine can only be obtained with a prescription.

  • Signifor (pasireotide)
     

Treatment of Cushing’s disease
Novartis Europharm Limited
United-Kingdom
24/04/2012
What is Signifor?
Signifor is a medicine that contains the active substance pasireotide. It is available as a solution for injection.
What is Signifor used for?
Signifor is used to treat adults with Cushing’s disease when surgery has failed or is not an option.
Cushing’s disease is caused by a tumour of the pituitary gland (a gland located at the base of the brain) releasing too much of a hormone called ACTH that stimulates the production of too much cortisol (a hormone also known as the ‘stress hormone’ because it is released in response to stress).
Because the number of patients with Cushing’s disease is low, the disease is considered ‘rare’, and Signifor was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.

  • Dacogen (decitabine)
     

acute myeloid leukaemia (AML)
Janssen-Cilag International NV, Belgium
20/09/2012
What is Dacogen?
Dacogen is a powder that is made up into a solution for infusion (drip into a vein). It contains the active substance decitabine.
What is Dacogen used for?
Dacogen is used to treat adults aged 65 or older with acute myeloid leukaemia (AML), a type of cancer affecting the white blood cells. It is used in patients with newly diagnosed AML who are not eligible for initial treatment with standard chemotherapy (anticancer medicines).
Because the number of patients with AML is low, the disease is considered ‘rare’, and Dacogen was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 June 2006.
The medicine can only be obtained with a prescription.
 
  • Revestive (teduglutide)
     

Short bowel syndrome
Nycomed Danmark APS
Denmark
30/08/2012

What is Revestive? 
Revestive is a medicine that contains the active substance teduglutide. It is available as a powder and a solvent to be made up into a solution for injection.
What is Revestive used for? 
Revestive is used to treat adults with short bowel syndrome. Short bowel syndrome is a condition in which nutrients and fluids are not properly absorbed by the gut, usually following the surgical removal of a large portion of the small intestine. Revestive is used after ‘intestinal adaptation’ has occurred (changes in the function of the bowel to compensate for its reduced size following surgery).
Because the number of patients with short bowel disease is low, the disease is considered ‘rare’, and Revestive was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 11 December 2001.
The medicine can only be obtained with a prescription.

  • Jakavi (ruxolitinib)
     

myelofibrosis
Novartis Europharm Limited, UK
United-Kingdom
23/08/2012

What is Jakavi? 
Jakavi is a medicine that contains the active substance ruxolitinib. It is available as tablets (5, 15 and 20 mg).
What is Jakavi used for? 
Jakavi is used to treat adults with myelofibrosis who have splenomegaly (enlarged spleen) or symptoms related to the disease such as fever, night sweats, bone pain and weight loss.
Myelofibrosis is a disease in which the bone marrow becomes very dense and rigid and produces abnormal, immature blood cells. Jakavi can be used in three types of the disease: primary myelofibrosis (also known as chronic idiopathic myelofibrosis, where the cause is unknown), post polycythaemia vera myelofibrosis (where the disease is linked to an overproduction of red blood cells) and post essential thrombocythaemia myelofibrosis (where the disease is linked to an overproduction of platelets, components that help the blood to clot).
Because the number of patients with these diseases is low, they are considered ‘rare’, and Jakavi was designated an ‘orphan medicine’ (a medicine used in rare diseases) for chronic idiopathic myelofibrosis on 7 November 2008 and for myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia on 3 April 2009.
The medicine can only be obtained with a prescription.

  • Kalydeco (ivacaftor)
     

cystic fibrosis
Vertex Pharmaceuticals (U.K.) Ltd.
United-Kingdom
23/07/2012

What is Kalydeco?
Kalydeco is a medicine that contains the active substance ivacaftor. It is available as tablets (150 mg).

What is Kalydeco used for?
Kalydeco is used to treat cystic fibrosis in patients aged six years and above who have the G551D mutation in their gene for the protein called cystic fibrosis transmembrane conductance regulator (CFTR). Cystic fibrosis is an inherited disease that affects the cells that secrete mucus in the lungs, and the cells that secrete digestive juices from the glands in the gut and pancreas. In cystic fibrosis these secretions become thick, blocking the airways and the flow of digestive juices. This leads to problems with the digestion and absorption of food, resulting in poor growth, and long-term infection and inflammation of the lungs because of excess mucus not being cleared away. Because the number of patients with cystic fibrosis is low, the disease is considered ‘rare’, and Kalydeco was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 July 2008.
The medicine can only be obtained with a prescription.

Latest marketing authorisations and orphan drug designations

Latest marketing authorisations and orphan drug designations



Marketing authorisations and medicinal products designations

Detailed information on European orphan medicinal products designation applications is available on theEMA website. A full list of designated and authorised orphan medicinal products in Europe available at:ec.europa.eu.

Orphan drug regulatory processLearn more about the Orphan designation process in Europe




Recent orphan drug designations

July 2014
Treatment of cystic fibrosis
1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide
Treatment of Usher syndrome
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of humanMYO7A gene and the 3'-half sequence of human MYO7A gene
Treatment of Stargardt's disease
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene
Treatment of invasive aspergillosis
Isavuconazonium sulfate
Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)
Afamelanotide
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Humanised Fc engineered monoclonal antibody against CD19
Treatment of Prader-Willi syndrome
Beloranib
Treatment of primary sclerosing cholangitis
Norursodeoxycholic acid
Treatment of pre-eclampsia
Recombinant human alpha-1-microglobulin
Treatment of choroideraemia
Adeno-associated viral vector serotype 2 containing the human REP1 gene
June 2014
Treatment of gastro-entero-pancreatic neuroendocrine tumours
Lutetium (177Lu) edotreotide
Treatment of biliary tract cancer
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-(-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9 tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one
Treatment of follicular lymphoma
177Lu-tetraxetan-tetulomab
Treatment of epidermolysis bullosa
Recombinant human alpha 1 chain homotrimer of type VII collagen
Treatment of glioma
Autologous dendritic cells pulsed with RNA from glioma stem cells
Treatment of glioma
Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe
Treatment of central retinal vein occlusion
Aganirsen
Treatment of mucormycosis
Isavuconazonium sulfate
Treatment of cystic fibrosis
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent
Treatment of choroideraemia
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
Prevention of scarring post glaucoma filtration surgery
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
Treatment of mucopolysaccharidosis IIIA (Sanfilippo A syndrome)
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
April 2014
Treatment of lymphoplasmacytic lymphoma
Ibrutinib
Treatment of ovarian cancer
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Treatment of B-lymphoblastic leukaemia/lymphoma
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Treatment of ATTR amyloidosis
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Treatment of plasma cell myeloma

Humanised monoclonal antibody against CD38
Prevention of bronchopulmonary dysplasia
Caffeine citrate
Prevention of bronchopulmonary dysplasia
Recombinant human surfactant protein D
March 2014
Treatment of ATTR amyloidosis
Phosphorothioate oligonucleotide targeted to transthyretin
Treatment of glycogen storage disease type II (Pompe's disease)
Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan
Treatment of pancreatic cancer
Cysteamine bitartrate
Prevention of graft rejection following solid organ transplantation
Ex-vivo-cultured human mesenchymal stromal cells
Prevention of graft rejection following solid organ transplantation
Eculizumab
Treatment of acute myeloid leukaemia
Volasertib
Treatment of Leber’s congenital amaurosis
Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency
Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Treatment of soft tissue sarcoma
Doxorubicin(6-maleimidocaproyl)hydrazone
Treatment of nontuberculous mycobacterial lung disease
Amikacin sulfate
Treatment of Charcot-Marie-Tooth disease type 1A
Fixed-dose combination of (R-S) baclofen, naltrexone hydrochloride and D-sorbitol
 
February 2014
Treatment of Farber disease
Recombinant human acid ceramidase
Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection
Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins
Treatment of epidermolysis bullosa
Diacerein
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Gallium [Ga-68]-N-[(4,7,10-tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(27)disulfide
Prevention of delayed graft function after solid organ transplantation
Eculizumab
Treatment of cystic fibrosis
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-azacyclopentadecane-13,15-dione
Treatment of cystic fibrosis
Cysteamine
Treatment of Duchenne muscular dystrophy
Asp-Arg-Val-Tyr-Ile-His-Pro
Treatment of Rett síndrome
3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl)amino]methyl}piperidin-1-yl]methanone
Treatment of polycythaemia vera
Ruxolitinib
Treatment of adrenoleukodystrophy
Pioglitazone
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid
Treatment of glioma
Autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha)
Treatment of optic neuritis
N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide
Treatment of familial chylomicronemia syndrome
Phosphorothioate oligonucleotide targeted to apolipoprotein CIII
January 2014
Treatment of malignant mesothelioma
Amatuximab
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Inecalcitol
Treatment of aneurysmal subarachnoid haemorrhage
Sodium nitrite
Treatment of hepatitis delta virus infection
Lonafarnib
Treatment of dystrophic myotonia
(6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4Hdibenzo[de,g]quinoline-2,9-diol
Treatment of adenovirus infection in allogeneic haematopoietic stem-cell transplant recipients
Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
Treatment of primary sclerosing cholangitis
Obeticholic acid
Treatment of malignant mesothelioma
Autologous dendritic cells pulsed with allogeneic tumour cell lysate
Treatment of acute myeloid leukaemia
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino) methyl)tetrahydrofuran-3,4-diol
Treatment of acute lymphoblastic leukaemia
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1Hbenzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl)amino)methyl)tetrahydrofuran-3,4-diol
Treatment of epidermolysis bullosa
Allantoin
Prevention of graft-versus-host disease
Allogeneic bone-marrow derived ex-vivo expanded multipotent adult progenitor cells
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino) phenyl)acrylamide benzenesulfonic acid salt
 
December 2013
Treatment of cystic fibrosis
Nitric oxide
Treatment of hypoparathyroidism
Recombinant human parathyroid hormone
Treatment of glioma
Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma
Treatment of follicular lymphoma
Ibrutinib
Prevention of necrotizing enterocolitis
Lactobacillus acidophilus and Bifidobacterium bifidum
Treatment of Alagille síndrome
 (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of primary biliary cirrosis
 (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of progressive familial intrahepatic cholestasis
 (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of primary sclerosing colangitis
 (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Prevention of arteriovenous access dysfunction in haemodialysis patients
Recombinant human type I pancreatic elastase
Treatment of Dravet syndrome
Fenfluramine hydrochloride
Treatment of dengue
Poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}ethyl]sulfanyl}-3-{[(2S)-1-ethoxy-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}propanamido)-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)propanoyl Ethyl ester) )-methoxy]acetyl}oxy)butyl)sulfanyl]-3-(hexadecylcarbamoyl)propanoic acid]-poly(ethylene glycol)-ester]
Treatment of haemophilia A
Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa
November 2013
Treatment of glioma
Autologous ex-vivo-expanded leucocytes treated with 5-aza-2’-deoxycytidine
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Recombinant human insulin receptor monoclonal antibody-fusediduronate 2-sulfatase
Treatment of follicular thyroid cancer
Sorafenib tosylate
Treatment of papillary thyroid cancer
Sorafenib tosylate
Prevention of graft-versus-host disease
Defibrotide
Treatment of hepatocellular carcinoma
Tivantinib
Treatment of diffuse large B-cell lymphoma
Ibrutinib
Prevention of arteriovenous access dysfunction in patients undergoing surgical creation of an arteriovenous access for haemodialysis
Sirolimus
Treatment of eosinophilic oesophagitis
Human monoclonal antibody against human interleukin 13
Treatment of spinal cord injury
Synthetic 12 amino acids peptide designed after subcommissural organ spondin
Treatment of ovarian cancer
Trebananib
Treatment of Stargardt’s disease
Soraprazan
October 2013
Treatment of congenital factor VII deficiency
Recombinant fusion protein linking coagulation factor VIIa with Albumin
Treatment of myotonic disorders
Mexiletine hydrochloride
Treatment of plasma cell myeloma
Recombinant human monoclonal IgM antibody targeting glucose-regulated protein 78
Treatment of sarcoidosis
L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-LSer-L-Ser
Treatment of complex regional pain syndrome
Zoledronic acid
Treatment of Allan-Herndon-Dudley syndrome
3,5-diiodothyropropionic acid
Treatment of Duchenne muscular dystrophy
Naproxcinod
Treatment of cystic fibrosis
Antisense oligonucleotide targeting the F508delta mutation of CFTR
Treatment of Wiskott-Aldrich-syndrome
Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene
July & August 2013
Treatment of pulmonary alveolar proteinosis
Granulocyte macrophage colony stimulating factor
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation
Human hemin
Treatment of B-lymphoblastic leukaemia/lymphoma
Moxetumomab pasudotox
Treatment of malignant thymomas
Belinostat
Treatment of pancreatic cancer
(1-methyl-2-nitro-1H-imidazole-5-yl) methyl N,N’-bis(2-bromoethyl) diamidophosphate
Treatment of plasma cell myeloma
Daratumumab
Treatment of ovarian cancer
Fosbretabulin tromethamine
Treatment of amyotrophic lateral sclerosis
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Prevention of hepatitis B re-infection following liver transplantation
Recombinant human monoclonal antibody against hepatitis B Virus
Treatment of follicular lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
Treatment of ataxia telangiectasia
Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes
Treatment of carbamoylphosphate synthase-1 deficiency
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 1
Heterologous human adult liver-derived progenitor cells
Treatment of argininosuccinic aciduria
Heterologous human adult liver-derived progenitor cells
Treatment of hyperargininaemia
Heterologous human adult liver-derived progenitor cells
Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 2
Heterologous human adult liver-derived progenitor cells
Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)
Heterologous human adult liver-derived progenitor cells
Treatment of limbal stem cell deficiency
Ex-vivo expanded autologous human corneal epithelium containing stem cells
Treatment of osteosarcoma
Lipid-complexed cisplatin
Treatment of acromegaly
Octreotide acetate (oral use)
Treatment of nodal marginal zone lymphoma
Idelalisib
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
Idelalisib
Treatment of acute myeloid leukaemia
Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Treatment of autosomal dominant polycystic kidney disease
Tolvaptan
Treatment of nontraumatic osteonecrosis
Human allogeneic bone marrow derived osteoblastic-like cells
Treatment of pancreatic cancer
Chimeric monoclonal antibody against claudin-18 splice variant 2
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab′antibody
Treatment of growth hormone deficiency
Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains
Treatment of Behçets' disease
Apremilast
Treatment of eosinophilic oesophagitis
Budesonide
Treatment of mastocytosis
Cladribine
Treatment of congenital sucrase-isomaltase deficiency
Sacrosidase
Treatment of sickle cell disease
(1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-2-yl)-β-D-galactopyranosyl]-4-O-(α-L-fucopyranosyl)-5-orothylamido-cyclohexane-1-carboxylic acid ethyl-2-amidyl-ethyloxy-2-acetyl-(8-amino-1,3,6-naphthalene-tris sodium sulfonate) amide
Treatment of neuromyelitis optica
Eculizumab
Treatment of extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)
Idelalisib
Treatment of splenic marginal zone lymphoma
Idelalisib
 
June 2013
Treatment of cystic fibrosis
4,6,4’–trimethylangelicin
Treatment of 5q spinal muscular atrophy
5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
Treatment of amyotrophic lateral sclerosis
Sodium chlorite
Treatment of retinitis pigmentosa
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina
Treatment of pachyonychia congenital
Synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation
Treatment of retinitis pigmentosa
Adenovirus associated viral vector serotype 5 containing the human pde6β gene
Treatment of acute liver failure
Immortalised human C3A hepatoblastoma cells
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Recombinant human alpha-N-acetylglucosaminidase
Treatment of soft tissue sarcoma
Genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor
Treatment of retinitis pigmentosa
Unoprostone isopropyl
Treatment of non-dystrophic myotonia
Mexiletine hydrochloride
Treatment of B-cell acute lymphoblastic leukaemia
Inotuzumab ozogamicin
Treatment of adrenocortical carcinoma
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
Treatment of graft-versus-host disease
Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
Treatment of transglutaminase-1-deficient autosomal recessive congenital ichthyosis
Recombinant human transglutaminase 1 encapsulated into liposomes
Treatment of cystic fibrosis
Recombinant human CXCL8 mutant
Treatment of malignant mesothelioma
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride
Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity
Maribavir
Treatment of adenosine deaminase-deficient-severe combined immunodeficiency
Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Treatment of retinitis pigmentosa
Recombinant human nerve growth factor
 
April 2013
Treatment of follicular thyroid cancer
Lenvatinib
Treatment of glioma
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of papillary thyroid cancer
Lenvatinib
Treatment of Duchenne muscular dystrophy
R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride
Treatment of idiopathic pulmonary fibrosis
Nintedanib
Treatment of Niemann-Pick disease, type C
2-hydroxypropyl-β-cyclodextrin
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
March 2013
Treatment of neuronal ceroid lipofuscinosis type 2
Recombinant human tripeptidyl-peptidase 1
Treatment of Stargardt’s disease
Ramiprilat
Treatment of Churg-Strauss Syndrome
Mepolizumab
Treatment of mantle cell lymphoma
1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H- pyrazolo [3,4-d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one
Treatment of congenital alpha-1 antitrypsin deficiency
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-Lglutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
Prevention of graft rejection following solid organ transplantation
Murine IgM monoclonal antibody binding to alpha beta T-cell receptor
Treatment of sickle cell disease
Poloxamer 188
Treatment of chronic non-infectious uveitis
Gevokizumab
Treatment of Niemann-Pick disease, type C
Recombinant human heat shock protein 70
Treatment of hepatocellular carcinoma
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of systemic sclerosis
2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid

February 2013
Treatment of achromatopsia caused by mutations in the CNGB3 gene
Recombinant adeno-associated viral vector containing the human CNGB3 gene
Treatment of amyloid light-chain amyloidosis
Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Treatment of moderate and severe traumatic brain injury
Progesterone
Treatment of high altitude pulmonary oedema
Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys
Treatment of chronic thromboembolic pulmonary hypertension
Treprostinil sodium
Treatment of systemic sclerosis
Terguride
Treatment of Duchenne muscular dystrophy
Humanised monoclonal antibody against myostatin
Treatment of acute myeloid leukaemia
L-asparaginase encapsulated in erythrocytes
January 2013
Treatment of follicular lymphoma
Lenalidomide
Treatment of familial adenomatous polyposis
Eflornithine in combination with sulindac
Treatment of growth hormone deficiency
Recombinant modified human growth hormone
Treatment of 5q spinal muscular atrophy
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Treatment of Wilson’s disease
Choline tetrathiomolybdate
Treatment of pancreatic cancer
Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen
Treatment of beta-thalassaemia intermedia and major
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Treatment of ovarian cancer
Chimeric monoclonal antibody against claudin 6
Treatment of glioma
1,2:5,6-Dianhydrogalactitol
Treatment of achondroplasia
Modified recombinant human C-type natriuretic peptide
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Treatment of systemic sclerosis
Terguride
Treatment of retinitis pigmentosa
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor