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


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


Treatment of chronic myeloid leukaemia 2005-12-23 Expired Sprycel


Treatment of progressive supranuclear palsy 2010-03-23 Withdrawn  

Deferoxamine mesilate

Treatment of traumatic spinal-cord injury 2004-10-20 Withdrawn  

Denufosol tetrasodium

Treatment of cystic fibrosis 2005-12-23 Withdrawn  

Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3')

Treatment of chronic lymphocytic leukaemia 2001-11-20 Withdrawn  

Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3')

Treatment of multiple myeloma 2001-11-20 Withdrawn  

Deuterium oxide

Treatment of pancreatic cancer 2004-10-20 Withdrawn  

Dexamethasone (40 mg tablet)

Treatment of multiple myeloma 2010-06-09 Withdrawn Neofordex

Dexamethasone (intravitreal implant)

Treatment of non-infectious uveitis affecting the posterior segment of the eye 2010-08-04 Withdrawn  

Dexamethasone phosphate (iontophoretic solution, ocular use)

Treatment of corneal graft rejection 2009-05-15 Withdrawn  


Treatment of anthracycline extravasations 2001-09-19 Expired Savene

Dihydroartemisinin, piperaquine

Treatment of malaria 2007-08-03 Withdrawn  


Treatment of alopecia totalis 2006-06-29 Withdrawn  


Treatment of alopecia universalis 2006-06-29 Withdrawn  

Doxorubicin carbon / iron magnetically targeted microparticles

Treatment of hepatocellular carcinoma 2002-09-11 Withdrawn  

Doxorubicin hydrochloride (drug eluting beads)

Treatment of glioma 2007-11-29 Withdrawn  

Dronabinol and cannabidiol

Treatment of glioma 2015-10-09 Withdrawn  

Ecteinascidin 743 (trabectedin)

Treatment of soft tissue sarcoma 2001-05-30 Expired Yondelis

Eflornithine hydrochloride

Treatment of familial adenomatous polyposis (FAP) 2002-02-19 Withdrawn  


Treatment of multiple myeloma 2012-08-09 Withdrawn  

Eltrombopag olamine

Treatment of idiopathic thrombocytopenic purpura 2007-08-03 Withdrawn Revolade

Engineered protein inhibitor of human neutrophil elastase

Treatment of cystic fibrosis 2003-07-09 Withdrawn  

Epothilone B

Treatment of ovarian cancer 2002-03-22 Withdrawn  

Eptacog alfa (activated)

Treatment of post-neonatal intracerebral haemorrhage 2007-03-19 Withdrawn  


Treatment of gastric cancer 2011-08-30 Withdrawn