Latest orphan designations and/or marketing authorisations
ORPHAN DRUG DESIGNATIONS
October 2012Treatment of cystic fibrosis
Alpha-1 proteinase inhibitor (for inhalation use)
Treatment of fragile X syndrome
Mavoglurant
Treatment of acute lung injury
Asp-Arg-Val-Try-Ile-His-Pro
Treatment of pancreatic cancer
Mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene
Treatment of ovarian cancer
Rucaparib
Treatment of traumatic spinal cord injury
[2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]
Treatment of lecithin cholesterol acyltransferase deficiency
Recombinant human lecithin cholesterol acyltransferase
Treatment of haemophilia A
Humanised monoclonal IgG4 antibody against tissue factor pathway inhibitor
Treatment of ovarian cancer
Lurbinectedin
Treatment of chronic lymphocytic leukaemia
Obinutuzumab
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Belinostat
Treatment of acute myeloid leukaemia
Liposomal daunorubicin
September 2012
Diagnosis of positive folate receptor status in ovarian cancer
N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-Lalpha-aspartyl-L-cysteine to be used with folic acid
Diagnosis of positive folate receptor status in ovarian cancer
Folic acid to be used with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-Lalpha-
aspartyl-L-cysteine
MARKETING AUTHORISATION
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Dacogen (decitabine)
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.
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Revestive (teduglutide)
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.
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Jakavi (ruxolitinib)
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.
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Kalydeco (ivacaftor)
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.
Detailed information on European orphan drug designation applications is available on the EMA website
A full list of designated and authorised orphan drugs in Europe available at: ec.europa.eu
Page created: 27/04/2012
Page last updated: 30/10/2012
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