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Table 2 EPARs included in the evaluation

From: Applicability and added value of novel methods to improve drug development in rare diseases

Cluster Drug Date opinion Rare or ultra-rare* Repurposed/ new drug?
Acute: single episodes
 Antracycline extravasation Savene 2006 Ultra-rare New drug
 Patent ductus arteriosus Pedea 2009 Rare Repurposed
 Hepatic venooclusive disease Defitelio 2013 Rare New drug
 Tuberculosis Sirturo 2014 Rare New drug
Acute: recurrent episodes
 Cryopirine periodic syndromes Ilaris 2009 Ultra-rare New drug
 Gram negative lung infection in cystic fibrosis Cayston 2009 Rare Repurposed
 Narcolepsy Xyrem 2007 Rare New drug
 Dravet syndrome Diacomit 2009 Rare New drug
 Sickle cell disease Sicklos 2007 Rare New drug
 Systemic sclerosis Tracleer 2009 Rare New drug
Chronic: stable/slow progression
 Short bowel syndrome Revestive 2012 Rare New drug
 Adrenal insufficiency Plenadren 2011 Rare Repurposed
 Thrombocytemia Xagrid 2009 Rare New drug
 Deficit of lipoprotein lipase Glybera 2012 Ultra-rare New drug
Chronic: progressive, one system/organ
 Nocturnal Paroxysmal haemoglobinuria Soliris 2009 Rare New drug
 Wilson’s disease Wilzin 2006 Rare New drug
 Congenital errors of bile synthesis Orphacol 2013 Ultra-rare Repurposed
 Gastrointestinal stromal tumours Glivec 2009 Ultra-rare Repurposed
Chronic: progressive, multiple systems/organs
 Fabry disease Fabrazyme 2008 Ultra-rare New drug
 Cystic fibrosis Kalydeco 2013 Rare New drug
 Familial amyloid polyneuropathy Vyndaqel 2011 Rare New drug
 Gaucher disease Zavesca 2009 Rare New drug
Chronic: staged condition
 Renal carcinoma Afinitor 2009 Rare Repurposed
 Pulmonary hypertension Opsumit 2014 Rare New drug
 Indolent non-Hodgkin lymphoma Litak 2006 Rare New drug
 Myelodysplastic syndrome Revlimid 2008 Rare New drug
  1. EPAR European Public Assessment Report
  2. * Rare if prevalence = or > 5/10.000 and > 0.1/10.000 inhabitants; Ultrarare if prevalence = or < 0.1/10.000 inhabitants