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How Many Orphan Drugs Did The FDA Approve of In 2012 ?

December 10, 2012

       According to a December 2012 FDA Report titled, “FY 2012 Innovative Drug Approvals” :

  1.  FDA approves 35 New Molecular Entities (NMEs) in its fiscal year 2012 (ending September)
  2. Of the 35 NMEs for 2012, 9 or approximately 26% are for orphan drugs
  3. In the last 5 years, approximately 1/3 of the NMEs are for rare diseases. 

       The 9 orphan drugs are listed in the chart below :   

Generic Name Trade Name  Sponsor Indication
Asparaginase   Erwinia Chrysanthemi Erwinaze EUSA Pharma Acute Lymphoblastic Leukemia (ALL)
Ruxolitinib Jakafi Incyte Corp Myelofibrosis
Ivacaftor Kalydeco Vertex Pharmaceuticals Cystic Fibrosis with G551D mutation
Glucarpidase Voraxaze BTG International Methotrexate Toxicity
Bosutinib Bosulif Pfizer Chronic Myelogenous Leukemia (CML)
Taliglucerase Alfa Elelyso Pfizer Type 1 Gaucher Disease
Deferiprone Ferriprox ApoPharma Iron overload due to Thalassemia Syndromes
Carfilzomib Kyprolis Onyx Therapeutics Multiple Myeloma
Clobazam Onfi Lundbeck Inc Seizures in Lennox-Gastaut Syndrome

Please note that the link for the “Indication” Column is the FDA Orphan Drug Product Designation Database Link 

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