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

1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one

Treatment of Stargardt's disease 2018-05-25 Positive  

Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII

Treatment of haemophilia A 2018-05-25 Positive  

Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles

Treatment of methylmalonic acidaemia 2018-05-25 Positive  

Adeno-associated viral vector serotype 9 containing the human CLN1 gene

Treatment of neuronal ceroid lipofuscinosis 2018-05-25 Positive  

Ambroxol hydrochloride

Treatment of amyotrophic lateral sclerosis 2018-05-25 Positive  

Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor

Treatment of follicular lymphoma 2018-05-25 Positive  

Bardoxolone methyl

Treatment of Alport syndrome 2018-05-25 Positive  


Treatment of AL amyloidosis 2018-05-25 Positive  

Equine immunoglobulin F(ab')2 fragments targeting Shiga toxin

Prevention of haemolytic uraemic syndrome 2018-05-25 Positive  

Glucagon analogue linked to a human immunoglobulin Fc fragment

Treatment of congenital hyperinsulinism 2018-05-25 Positive  


Treatment of glioma 2018-05-25 Positive  


Prevention of invasive aspergillosis 2018-05-25 Positive  

Three human monoclonal antibodies against the EBOV glycoprotein

Treatment of Ebola virus disease 2018-05-25 Positive  

synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues

Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis) 2018-05-25 Positive  

Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene

Treatment of glycogen storage disease type II (Pompe's disease) 2018-04-16 Positive  

adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1

Treatment of amyotrophic lateral sclerosis 2018-04-16 Positive  

autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo

Treatment of ovarian cancer 2018-04-16 Positive  


Treatment of spinal muscular atrophy 2018-04-16 Positive  


Treatment of phosphaturic mesenchymal tumour 2018-04-16 Positive  

genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII

Treatment of epidermolysis bullosa 2018-04-16 Positive  

polatuzumab vedotin

Treatment of diffuse large B-cell lymphoma 2018-04-16 Positive  


Treatment of C3 glomerulopathy 2018-03-21 Positive  

Dimethyl fumarate

Treatment of Friedreich's ataxia 2018-03-21 Positive  

Docosahexaenoic acid ethyl ester

Treatment of sickle cell disease 2018-03-21 Positive  

Efgartigimod alfa

Treatment of myasthenia gravis 2018-03-21 Positive