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Orphan Drugs And Rare Diseases: Review #2 Of 2013

July 9, 2013

This is the second in a series of Blog Posts over the next two weeks on the review of the first six months of 2013 in the orphan drug and rare disease space.

The chart below compares the number of FDA Orphan Drug Designation (ODD) Approvals in the first 6 months of 2012 versus the number of FDA ODD Approvals in the first 6 months of 2013. The FDA’s Orphan Drug Product Designation Database Application is the source of the information.

# of Jan-Jun   2012 FDA ODD Approvals

# of Jan-Jun   2013 FDA ODD Approvals

% Change From 2012 To 2013



88.8 %

Thus, there is approximately a 89% increase in the # of FDA ODD Approvals in the first 6 months of 2013 compared to the # of FDA ODD Approvals in the first 6 months of 2012.

The chart below shows the 17 FDA ODD Approvals in the first 6 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

Please Note: “A group of three balloons on ribbons” JPEG by AJ (Open clip Art Library image’s page) [see page for license] | Wikimedia Commons.

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

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