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FDA Orphan Drug Approvals: 6-Months In Review

July 7, 2014


This is the second in a series of Blog Posts on the review of the first six months of 2014 for orphan drugs and rare diseases. This Blog Post will compare the number of FDA Orphan Drug Approvals of the first 6 months of 2014 versus the number of FDA Orphan Drug Approvals of the first 6 months of 2013.

First 6-Months Of FDA Orphan Drug Approvals For 2013 & 2014


# of Jan-Jun 2013 FDA Orphan Drug Approvals # of Jan-Jun 2014 FDA Orphan Drug Approvals % Change
19 17 10.5% –


List of FDA Orphan Drug Approvals For First 6-Months Of 2014

# Brand/ Generic Name Approval Indication Approval Date Sponsor Company
1 Trametinib and Dabrafenib/ Mekinist and Tafinlar In Combination for Unresectable/Metastatic Melanoma with BRAF V600E or V600K mutations 01-08-2014 GSK
2 Tasimelteon/ Hetlioz Non-24-Hour Sleep-Wake Disorder 01-31-2014 Vanda Pharmaceuticals
3 Ibrutinib/ Imbruvica CLL for those who receive at least 1 prior therapy 02-12-2014 Pharmacyclics
4 Elosulfase Alfa/ Vimizim Mucopolysaccharidosis (MPS) Type IV A (Morquio A Syndrome) 02-14-2014 BioMarin Pharmaceutical
5 Droxiopa/ Northera Symptomatic Neurogenic Orthostatic Hypotension 02-18-2014 Lundbeck LLC
6 Metreleptin/ Myalept Lipodystrophy 02-24-2014 Amylin Pharmaceuticals
7 Propranolol/ Hemangeol Proliferating Infantile Hemangiomas 03-14-2014 Pierre Fabre Dermatologie
8 Miltefosine/ Impavido Leishmaniasis 03-19-2014 Paladin Therapeutics
9 Coagulation factor IX (recombinant), Fc fusion protein/ Alprolix Hemophilia B to reduce frequency of bleeding episodes 03-28-2014 Biogen Idec
10 Ecallantide/ Kalbitor Acute attacks of Hereditary Angioedema (HAE) 03-28-2014 Dyax Corp.
11 Ethiodized Oil Injection/ Lipiodol Selective hepatic intra-arterial use for imaging tumors 04-04-2014 Guerbet LLC
12 Ofatumumab/ Arzerra Ofatumumab in combination with chlorambucil, for previously untreated CLL 04-17-2014 Glaxo Group Limited d/b/a GSK
13 Ramucirumab/ Cyramza Advanced gastric cancer/gastro-esophageal junction adenocarcinoma 04-21-2014 Eli Lilly & Co.
14 Siltuximab/ Sylvant Castleman’s Disease 04-23-2014 Janssen Biotech
15 Mercaptopurine Oral Solution/ Purixan Acute lymphoblastic leukemia as part of a combination regimen 04-28-2014 Nova Laboratories Limited (UK)
16 Ceritinib/ Zykadia ALK+ NSCLC 04-29-2014 Novartis Pharmaceuticals
17 Antihemophilic factor (recombinant), Fc fusion protein Hemophilia A to reduce frequency of bleeding episodes 06.06.2014 Biogen Idec



•   Slight decrease of 10% of the # of FDA Orphan Drug Approvals for the first 6-months of 2014 compared to 2013

•   Biogen Idec is the big winner for the first 6 months of 2014 with 2 FDA Orphan Drug Approvals, which continues the company’s Hemophilia A and B franchise

•   Approval of Janssen Biotech’s Sylvant for the rare disease, Castleman’s Disease

•   Two FDA approvals for Chronic Lymphocytic Leukemia CLL

•   Approval of BioMarin’s Vimizim for the rare disease, Morquio A Syndrome

•   Approximately 35% of the FDA approvals are for an oncology indication.

Please Note: “LuMaxArt Golden Guys Teamwork Gear Runners” by lumaxart [CC-BY-SA-2.0] | Wikimedia Commons..

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