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

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 orphandruganaut@gmail.com 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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