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

Iloprost

Treatment of primary and of the following forms of secondary pulmonary hypertension… 2000-12-29 Expired Ventavis

Imatinib mesilate

Treatment of dermatofibrosarcoma protuberans 2005-08-26 Expired Glivec

Imatinib mesilate

Treatment of mastocytosis 2005-08-26 Withdrawn  

Imatinib mesilate

Treatment of acute lymphoblastic leukaemia 2005-08-26 Expired Glivec

Imatinib mesilate

Treatment of chronic eosinophilic leukaemia and the hypereosinophilic syndrome 2005-10-28 Expired Glivec

Imatinib mesilate

Treatment of chronic myeloid leukaemia 2001-02-14 Expired Glivec

Imatinib mesilate

Treatment of malignant gastrointestinal stromal tumours 2001-11-20 Expired Glivec

Imatinib mesilate

Treatment of myelodysplastic / myeloproliferative diseases 2005-12-23 Expired Glivec

Interferon gamma

Treatment of idiopathic pulmonary fibrosis 2005-05-27 Withdrawn  

Iodine (131I) anti-tenascin monoclonal antibody 81C6

Treatment of glioma 2006-10-29 Withdrawn  

Iodine (131I) Anti-CEA sheep-human chimeric monoclonal antibody

Treatment of pancreatic cancer 2003-05-08 Withdrawn  

Iodine (131I) anti-nucleohistone H1 chimeric biotinylated monoclonal antibody

Treatment of glioma 2002-11-12 Withdrawn  

Iodine (131I) chimeric IgG monoclonal antibody cG250

Treatment of renal cell carcinoma 2002-03-19 Withdrawn  

Iodine (131I) tositumomab

Treatment of follicular lymphoma 2003-02-13 Withdrawn  

Irinotecan hydrochloride (drug-eluting beads)

Treatment of glioma 2007-11-29 Withdrawn  

Kifunensine

Treatment of alpha sarcoglycanopathy 2011-09-27 Withdrawn  

Kifunensine

Treatment of beta sarcoglycanopathy 2011-09-27 Withdrawn  

Kifunensine

Treatment of delta sarcoglycanopathy 2011-09-27 Withdrawn  

Kifunensine

Treatment of gamma sarcoglycanopathy 2011-09-27 Withdrawn  

L-Asparaginase

Treatment of acute lymphoblastic leukaemia 2005-01-26 Withdrawn  

Lentiviral vector containing the human Wiskott Aldrich Syndrome Protein gene

Treatment of Wiskott Aldrich syndrome 2006-01-24 Withdrawn  

Lestaurtinib

Treatment of acute myeloid leukaemia 2006-07-25 Withdrawn  

Levofloxacin hemihydrate

Treatment of cystic fibrosis 2008-09-23 Withdrawn  

Liarozole

Treatment of congenital ichthyoses 2003-06-10 Withdrawn  

Linsitinib

Treatment of adrenal cortical carcinoma 2012-04-02 Withdrawn