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FDA Orphan Drug Designations: 2013 9-Month Approval Rate

October 8, 2013

This is the second in a series of Blog Posts this week on the review of the first nine months of 2013 for orphan drugs.

The chart below compares the number of FDA Orphan Drug Designation (ODD) Marketing Approvals in the first 9 months of 2012 versus the number of FDA ODD Marketing Approvals in the first 9 months of 2013. The FDA’s Orphan Drug Product Designation Database Application is the source of the information.

# of Jan-Sept   2012   FDA ODD Approvals

# of Jan-Sept  2013   FDA ODD Approvals

% Change From 2012 To   2013



61.5 %

Thus, there is approximately a 62 % increase in the # of FDA ODD Marketing Approvals in the first 9 months of 2013 compared to the # of FDA ODD Marketing Approvals in the first 9 months of 2012.

The chart below shows the 21 FDA ODD Marketing Approvals in the first 9 months of 2013, in ascending chronological order:

Brand/Generic    Name

Orphan   Designation

Marketing   Approval   Date


                                                                               1)   Kynamro (Mipomersen) Homozygous Familial   Hypercholesterolemia (HoFH) 01-29-2013 Genzyme
      2) Ravicti (Glycerol   Phenylbutyrate) Urea Cycle Disorder   (UCD) 02-01-2013 Hyperion   Therapeutics
      3) Pomalidomide (Pomalidomide) Multiple Myeloma   (MM) 02-08-2013 Celgene
      4)   Stivarga (Regorafenib) Gastrointestinal   Stromal Tumor (GIST) 02-25-2013 Bayer
      5) Gammaplex (Human   Immunoglobulin) Immune   Thrombocytopenic Purpura (ITP) 03-08-2013 Bio Products Labs   (BPL), UK
      6) Botulism Antitoxin   Heptavalent   (A-G) (Equine) Botulism 03-22-2013 Cangene Corp,   Canada
      7) Actemra (Tocilizumab) Juvenile Idiopathic   Arthritis 04-29-2013 Genentech
      8) Kcentra (Prothrombin   Complex Concentrate) Reversal of   Acquired Coagulation Factor Deficiency Induced   By Vitamin K Antagonist   Therapy for Acute Major Bleeding 04-29-2013 CSL Behring
      9)   Procysbi (Cysteamine Enteric Coated) Nephropathic    Cystinosis 04-30-2013 Raptor Therapeutics
      10)   llaris (Canakinumab) Systemic Juvenile   Idiopathic Arthritis (SJIA) 05-09-2013 Novartis
      11)   Nymalize (Nimodipine) Subarachnoid   Hemorrhage (SAH) 05-10-2013 Arbor Pharma
      12) Ixiaro (Japanese   Encephalitis Vaccine) Prevention of   Japanese Encephalitis Virus 05-17-2013 Intercell AG,   Austria
      13)   Tafinlar (Dabrafenib) BRAF V600E   Mutation Positive Melanoma 05-29-2013 GSK
14)      Mekinist (Trametinib) BRAF V600E or   V600K  Mutation Positive Melanoma 05-29-2013 GSK
      15)   Revlimid (Lenalidomide) Mantle Cell   Lymphoma 06-05-2013 Celgene
      16) Xgeva (Denosumab) Giant Cell Tumor Of   Bone 06-13-2013 Amgen
      17) Rixubis (Coagulation   Factor IX Recombinant) Hemophilia B 06-26-2013 Baxter
 18) Afatinib  EGFR+ NSCLC  07-12-2013  Boehringer Ingelheim
 19) enalapril maleate (powder for oral solution)  Hypertension  08-13-2013  Silvergate Pharma
 20) Meclorethamine  Mycosis Fungoides  08-23-2013  Ceptaris Therapeutics
 21) paclitaxel protein-bound particles  Pancreatic Cancer  09-06-2013  Abraxis BioScience


Please Note: “Interferon” courtesy of Linda Bartlett (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].

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

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