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

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Treatment of cystic fibrosis 2008-09-23 Withdrawn  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Treatment of congenital ichthyoses 2003-06-10 Withdrawn  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Treatment of adrenal cortical carcinoma 2012-04-02 Withdrawn  

Lintuzumab

Treatment of acute myeloid leukaemia 2009-04-30 Withdrawn  

Lintuzumab

Treatment of myelodysplastic syndrome 2009-04-29 Withdrawn  

Lumacaftor / ivacaftor

Treatment of cystic fibrosis 2014-08-22 Withdrawn Orkambi

Lumiliximab

Treatment of chronic lymphocytic leukemia 2008-02-03 Withdrawn  

Lusupultide

Treatment of acute respiratory distress syndrome (ARDS) 2001-01-16 Withdrawn  

Lusupultide

Treatment of aspiration pneumonitis requiring intubation and mechanical ventilation 2007-07-10 Withdrawn  

Maytansinoid-conjugated humanised monoclonal antibody against CD56

Treatment of Merkel-cell carcinoma 2010-06-09 Withdrawn  

Maytansinoid-conjugated humanised monoclonal antibody against CD56

Treatment of multiple myeloma 2011-02-23 Withdrawn  

Maytansinoid-conjugated humanised monoclonal antibody against CD56

Treatment of small-cell lung cancer 2010-10-01 Withdrawn  

Mazindol

Treatment of narcolepsy 2015-02-12 Withdrawn  

Mecasermin rinfabate

Treatment of growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH 2006-06-20 Withdrawn  

Mecasermin rinfabate

Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects 2006-06-20 Withdrawn  

Mecasermin

Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects 2006-05-22 Expired Increlex

Melatonin

Treatment of non-24-hour sleep-wake disorders in blind people with no light perception 2005-04-11 Withdrawn  

Mifepristone

Treatment of Cushing's syndrome secondary to ectopic ACTH secretion 2005-07-27 Withdrawn  

Mitotane

Treatment of adrenal cortical carcinoma 2002-06-12 Expired Lysodren

Mitotane

Treatment of adrenal cortical carcinoma 2002-09-11 Withdrawn  

Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins

Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection 2014-02-20 Withdrawn  

Mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene

Treatment of pancreatic cancer 2012-10-10 Withdrawn  

Monoclonal antibody to human interleukin-6

Treatment of post-transplantation lymphoproliferative disorders 2002-12-18 Withdrawn  

Murine IgM monoclonal antibody binding to alpha beta T-cell receptor

Prevention of graft rejection following solid organ transplantation 2013-03-12 Withdrawn  

Murine anti-idiotypic antibody against OC125 antibody against CA125 antigen

Treatment of ovarian cancer 2003-07-09 Withdrawn