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


Treatment of gastro-entero-pancreatic neuroendocrine tumours 2007-11-13 Withdrawn  


Treatment of renal cell carcinoma 2007-06-05 Withdrawn Afinitor


Treatment of primary malignant bone tumours 2007-01-25 Withdrawn  


Treatment of soft tissue sarcoma 2007-01-29 Withdrawn  


Treatment of chronic inflammatory demyelinating polyneuropathy 2010-02-02 Withdrawn  


Treatment of glioblastoma 2000-10-18 Withdrawn  

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-L-alpha-aspartyl-L-cysteine

Diagnosis of positive folate-receptor status in ovarian cancer 2012-09-10 Withdrawn Neocepri


Treatment of methanol poisoning 2001-05-30 Withdrawn  


Treatment of diarrhoea associated with intestinal microsporidial infection 2002-02-04 Withdrawn  

GM-CSF receptor antagonist

Treatment of juvenile myelomonocytic leukaemia 2002-03-18 Withdrawn  


Treatment of Schnitzler syndrome 2014-08-22 Withdrawn  


Treatment of chronic non-infectious uveitis 2013-03-12 Withdrawn  


Treatment of systemic-onset juvenile idiopathic arthritis 2010-01-28 Withdrawn  

Glutathione-pegylated liposomal doxorubicin hydrochloride

Treatment of glioma 2010-09-20 Withdrawn  


Treatment of cystic fibrosis 2006-04-11 Withdrawn  

H-Tyrosine-Glycine-Phenylalanine-Glycine-Glycine-OH (Chemical)

Treatment of chronic idiopathic myelofibrosis 2003-10-20 Withdrawn  


Treatment of Myasthenia Gravis 2006-08-28 Withdrawn  

HLA class I/II binding tumour associated peptides (ADF-APO-CCN-GUC-K67-MET-MMP-MUC-RGS)

Treatment of renal cell carcinoma 2007-02-14 Withdrawn  

Heparin sodium (inhalation use)

Treatment of cystic fibrosis 2005-12-22 Withdrawn  


Treatment of idiopathic pulmonary fibrosis 2004-09-02 Withdrawn  


Treatment of acute myeloid leukaemia 2004-10-20 Withdrawn  


Treatment of chronic myeloid leukaemia 2004-09-02 Withdrawn  

Human C1 inhibitor

Treatment of angioedema caused by C1 inhibitor deficiency 2009-10-08 Withdrawn  

Human Milk Fat Globule 1 / Yttrium (90Y) human Milk Fat Globule 1 - S p isothiocyanatobenzyl-diethylenetriaminepentaacetic acid

Treatment of ovarian cancer 2001-05-30 Withdrawn  

Human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin

Late onset sepsis in premature infants of less or equal than 32 weeks of gestational age 2005-12-13 Withdrawn