Rare disease (orphan) designations

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This page allows you to find information on rare disease (orphan) designations based on applications that have been assessed by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP).

An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

For more information, see Orphan designation.

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EPAR Search results
Active substance Disease / condition Date of decision Decision Medicine name

2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione

Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes 2017-12-12 Positive  

4-hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl

Treatment of familial cerebral cavernous malformation 2017-12-12 Positive  

Pegunigalsidase alfa

Treatment of Fabry disease 2017-12-12 Positive  

Venetoclax

Treatment of mantle cell lymphoma 2017-12-12 Positive  

(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide

Treatment of paroxysmal nocturnal haemoglobinuria 2017-12-12 Positive  

Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene

Treatment of Leber's congenital amaurosis 2017-12-12 Positive  

Modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles

Treatment of argininosuccinic aciduria 2017-12-12 Positive  

Acetylleucine

Treatment of GM2 gangliosidosis 2017-12-12 Positive  

Agammaglobulinaemia tyrosine kinase

Treatment of pemphigus 2017-12-12 Positive  

2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine

Treatment of Charcot-Marie-Tooth disease 2017-10-16 Positive  

5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]

Treatment of biliary tract cancer 2017-10-16 Positive  

Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters

Treatment of pancreatic cancer 2017-10-16 Positive  

Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells

Treatment of multiple myeloma 2017-10-16 Positive  

Entospletinib

Treatment of acute myeloid leukaemia 2017-10-16 Positive  

Glucopyranosyl lipid A

Treatment of follicular lymphoma 2017-10-16 Positive  

Ofranergene obadenovec

Treatment of ovarian cancer 2017-10-16 Positive  

Seladelpar

Treatment of primary biliary cholangitis 2017-10-16 Positive  

Siplizumab

Treatment in solid organ transplantation 2017-10-16 Positive  

Bitopertin

Treatment of beta-thalassaemia intermedia and major 2017-10-16 Positive  

Cannabidiol

Treatment of West syndrome 2017-10-16 Positive  

Cannabidivarin

Treatment of Rett syndrome 2017-10-16 Positive  

Glasdegib maleate

Treatment of acute myeloid leukaemia 2017-10-16 Positive  

Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F

Treatment of multiple myeloma 2017-10-16 Positive  

Pracinostat

Treatment of acute myeloid leukaemia 2017-10-16 Positive  

Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene

Treatment of Leber’s congenital amaurosis 2017-10-16 Positive