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Upcoming January – April 2013 FDA PDUFA Dates For Orphan Drugs (January 17, 2013 Update)

January 16, 2013
Generic Name Trade Name Sponsor Indication PDUFA Date
Glycerol Phenylbutyrate Ravicti Hyperion    Therapeutics ** Urea Cycle Disorder (UCD) 1/23/13
Mipomersen Sodium Kynamro Genzyme/Isis   Pharmaceuticals Homozygous Familial Hypercholesterolemia (HoFH) 1/29/13
Ampligen Hemispherx BioPharma Chronic Fatigue Syndrome (CFS) 2/02/13
Pomalidomide Celgene Corporation Relapsed refractory multiple myeloma 2/10/13
Regorafenib Stivarga Bayer GastroIntestinal Stromal Tumors (GIST) 2/28/13
Ponatinib Ariad   Pharmaceuticals Chronic Myeloid Leukemia & Acute Lymphoblastic Leukemia 3/27/13
Cysteamine Bitartrate Procysbi Raptor   Pharmaceutical Nephropathic Cystinosis 4/30/13

**Update : Per 01/16/13 Hyperion Therapeutics’ Press Release, FDA has advised them not to expect a final action by the PDUFA date of 01/23/13, because the FDA is “continuing to work on label & post-marketing requirements”.

Please notify at if any updates are available.

Please note the links for the following 2 chart columns above :

1) “Generic Name” Column Link = Is a source for the FDA PDUFA Date

2) “Indication” Column Link = Is the FDA Orphan Drug Product Designation Database Link

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