Rare disease (orphan) designations

  • Email
  • Help

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.

Document status

Include:

Browse by letter

Search for active substance by letter and/or number:

Search by keyword

Search for keyword:

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

Alpha-1-acid glycoprotein

Treatment of cocaine poisoning 2009-10-08 Withdrawn  

Alpha-1-acid glycoprotein

Treatment of tricyclic anti-depressants poisoning 2003-03-20 Withdrawn  

Alpha-galactosidase A

Treatment of Fabry disease 2000-08-08 Expired Replagal

Alpha-galactosidase A

Treatment of Fabry disease 2000-08-08 Expired Fabrazyme

Ambrisentan

Treatment idiopathic pulmonary fibrosis 2010-10-01 Withdrawn  

Amiloride hydrochloride dihydrate

Treatment of cystic fibrosis 2003-06-30 Withdrawn  

Amonafide L-malate

Treatment of acute myeloid leukaemia 2007-10-22 Withdrawn  

Anagrelide hydrochloride

Treatment of essential thrombocythaemia 2000-12-29 Expired Xagrid

Anti-CD 147 murine monoclonal IgM

Treatment of steroid resistant graft versus host disease (GvHD) 2002-11-13 Withdrawn  

Anti-von Willebrand aptamer

Treament of thrombotic thrombocytopenic purpura 2008-06-03 Withdrawn  

Aplidine

Treatment of acute lymphoblastic leukaemia 2003-07-09 Withdrawn  

Apomorphine (oromucosal use)

Treatment of off-periods in Parkinson’s disease not responding to other oral treatment 2001-12-04 Withdrawn  

Arsenic trioxide

Treatment of acute myeloid leukaemia 2007-08-02 Withdrawn  

Arsenic trioxide

Treatment of acute promyelocytic leukaemia 2000-10-18 Expired Trisenox

Arsenic trioxide

Treatment of acute promyelocytic leukaemia 2001-01-16 Withdrawn  

Arsenic trioxide

Treatment of multiple myeloma 2001-03-29 Withdrawn  

Arsenic trioxide

Treatment of myelodysplastic syndromes 2001-03-29 Withdrawn  

Ataluren

Treatment of mucopolysaccharidosis type I 2014-11-19 Withdrawn  

Autologous CD34+ cells transduced with retroviral vector containing the human gp91 (phox) gene

Treatment of chronic granulomatous disease 2006-07-13 Withdrawn  

Autologous urothelial and smooth muscle cells

Treatment of spina bifida 2008-03-17 Withdrawn  

Autologous urothelial and smooth muscle cells

Treatment of spinal cord injury 2008-11-07 Withdrawn  

Axitinib

Treatment of renal-cell carcinoma 2011-02-23 Withdrawn Inlyta

Azacitidine

Treatment of nasopharyngeal carcinoma 2015-11-11 Withdrawn  

Bafetinib

Treatment of chronic myeloid leukaemia 2010-06-10 Withdrawn  

Beraprost sodium

Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension 2001-09-18 Withdrawn