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Status Update On Upcoming 2012 FDA PDUFA Dates For Orphan Drugs

December 15, 2012

The Orphan Druganaut Blog has the following updates to its November 25, 2012 Post‘s chart on remaining 2012 FDA PDUFA Dates for orphan drugs :

Generic Name Trade Name Sponsor Indication PDUFA Date
Cabozantinib Exelixis Medullary Thyroid Cancer (MTC) 11/29/12
Raxibacumab GSK Anthrax 12/15/12
Lomitapide Aegerion Pharmaceuticals Homozygous Familial Hypercholesterolemia (HoFH) On or about 12/28/12
Teduglutide Gattex NPS Pharmaceuticals Short   Bowel Syndrome (SBS) On or about 12/28/12

(1)    Cabozantinib :

Here is the sequence of historical events for the FDA approval of Cometriq :

a) Receives FDA Orphan Drug Designation (ODD) on November 29, 2010

b) Submits Rolling New Drug Application (NDA) in May 2012

c) Receives FDA Priority Review on July 30, 2012 and assigning of November 29, 2012, for the PDUFA date

d) Removal from FDA Oncologic Drug Advisory Committee (ODAC) Meeting scheduled for November 8 – 9, 2012, on August 29, 2012

*** e) Approval on PDUFA date of November 29, 2012.

(2)   Raxibacumab :

FDA announces on December 14, 2012, that the drug is approved to treat inhalational anthrax. It is the 1st monoclonal antibody approved using the Animal Efficacy Rule, “which allows efficacy findings from adequate and well-controlled animal studies to support FDA approval when it is not feasible or ethical to conduct trials in humans. In this case, because inhalational anthrax is a rare and lethal disease, it is not possible to conduct adequate efficacy trials in humans.”   Raxibacumab was granted FDA fast track designation, priority review, and orphan product designation.

(3)   Lomitapide :

FDA decision will come on or before Friday, December 28, 2012 (per video reference below).

(4)   Teduglutide :

FDA decision will come on or before Friday, December 28, 2012 (per video reference below).

References December 14, 2012 Video titled, “The Next Big Thing in BioTech: FDA Decisions”.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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