Latest orphan designations and/or marketing authorisations
ORPHAN DRUG DESIGNATIONS
June 2013Treatment of cystic fibrosis
4,6,4’–trimethylangelicin
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
December 2012
Treatment of lead toxicity
Erdosteine
Treatment of malignant mesothelioma
Maytansinoid-conjugated human monoclonal antibody against mesothelin
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Alisertib
Treatment of acromegaly
Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-Dtryptophanyl-
L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt
Treatment of perinatal asphyxia
Allopurinol sodium
Treatment of Duchenne muscular dystrophy
Exon 52 specific phosphorothioate oligonucleotide
Treatment of Duchenne muscular dystrophy
Exon 55 specific phosphorothioate oligonucleotide
Treatment of malaria
Artesunate
Treatment of chronic lymphocytic leukaemia
4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide
Treatment of very-long-chain-acyl-CoA dehydrogenase deficiency
Triheptanoin
Treatment of long-chain L-3-hydroxyacyl-CoA-dehydrogenase deficiency
Triheptanoin
Treatment of chronic lymphocytic leukaemia
Humanised single chain monoclonal antibody against CD37
Treatment of non-infectious uveitis
Voclosporin
November 2012
Treatment of mercury toxicity
Erdosteine
Treatment of cutaneous T-cell lymphoma
Naloxone hydrochloride dehydrate
Treatment of glioma
IL-12-secreting dendritic cells, loaded with autologous tumour lysate
Treatment of malignant thymoma
Milciclib maleate
Treatment of systemic light chain amyloidosis
Ixazomib
Treatment of idiopathic pulmonary fibrosis
Tralokinumab
Treatment of neuroblastoma
Chimeric monoclonal antibody against GD2
Treatment of multiple myeloma
Panobinostat
Treatment of ovarian cancer
Alisertib
Treatment of glioma
Synthetic double-stranded siRNA oligonucleotide directed against claudin-5 complexed with polyethyleneimine (prior to administration of doxorubicin)
Treatment of senile systemic amyloidosis
tafamidis
Treatment of African tripanosomiasis
Melarsoprol
Treatment of retinitis pigmentosa
Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17- dimethylaminoethylamino-17-demethoxygeldanamycin)
Treatment of dyskeratosis congénita
Recombinant human dyskerin
Treatment of tumour necrosis factor receptor-associated periodic syndrome
Canakinumab
Treatment of macular telangiectasia type 2
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
October 2012
Treatment 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
August 2012
Treatment of primary biliary cirrhosis
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid
MARKETING AUTHORISATION
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Bosulif [bosutinib]
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.
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NexoBrid (concentrate of proteolytic enzymes enriched in bromelain)
Teva Pharma GmbH
Germany
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.
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Glybera (alipogene tiparvovec)
uniQure biopharma B.V.
The Netherlands
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.
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Adcetris ( brentuximab vedotin)
Takeda Global Research and Development Centre (Europe) Ltd
United Kingdom
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.
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Signifor (pasireotide)
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.
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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: 03/07/2013
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