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FDA March 2014 Products Receiving Orphan Designation And Approval

April 1, 2014




The chart below identifies FDA March 2014 Products Receiving Orphan Designation as of 03/31/14 in ascending “Orphan Drug Designation Date” order.

FDA March 2014 Products Receiving ODD

 #   Generic Name/ODD Date Sponsor Company Indication
1 Fecal Microbiota/ 03.10 Rebiotx Inc. Recurrent Clostridium Difficile Infection
2 Adeno Associated Virus with modified transthyretin and sequence encoding factor IX variant gene/ 03.14 Chatham Therapeutics Hemophilia B
3 Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19/ 03.14 Novartis Pharmaceuticals Chronic Lymphocytic Leukemia
4 Recombinant Human collagen alpha-1 (VII) chain homo-trimer (rC7)/ 03.17 Shire Human Genetic Therapeutics Dystrophic Epidermolysis Bullosa
5 (RS)-baclofen, naltrexone and D-sorbitol/ 03.17 Pharnext SAS (France) Charcot-Marie-ToothDisease type 1A
6 Ad5/3-D24-granulocyte-macrophage colony stimulating factor (GMCSF)-encoding oncolytic adenovirus/ 03.17 Oncos Therapeutics (Finland) Ovarian Cancer
7 Lenvatinib/ 03.26 Eisai Stage IIB to Stage IV Melanoma
8 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide/ 03.27 AbbVie Diffuse Large B-Cell Lymphoma
9 L-asparaginase/ 03.27 Erytech Pharma (France) Acute Myeloid Leukemia
10 Laromustine/ 03.27 Nanotherapeutics Acute Myelogenous Leukemia
11 autologous peripheral blood T lymphocytes transduced with retroviral vector containing anti CD19 CD28/CD3 zeta chimeric antigen receptor/ 03.27 Kite Pharma Diffuse Large B-Cell Lymphoma
12 Lenvatinib/ 03.27 Eisai HepatocellularCarcinoma


** “Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Some Observations

•   12 products receive orphan designation in March 2014 (as of 03/31/14)

•   2 products receive orphan designation for Diffuse Large B-Cell Lymphoma

•   Pharnext (France) receives orphan designation for rare disease Charcot-Marie-Tooth disease type 1A

•  2/3 of the March 2014 orphan designations are for an oncology indication

•  Chatham Therapeutics receives orphan designation for treatment of Hemophilia B.

FDA March 2014 Orphan Drugs Receiving Approval

The chart below identifies orphan drug designated products receiving FDA approval in March 2014 (as of 03/31/14) in ascending “Approval Date” order.


#   Generic Name/Approval Date Sponsor Company Indication
1 Propranolol (Hemangeol)/ 03.14 Pierre Fabre Dermatologie Proliferating Infantile Hemangioma
2 Miltefosine (Impavido)/ 03.19 Paladin Therapeutics Leishmaniasis
3 Coagulation factor IX (recombinant), Fc fusion protein (Alprolix)/ 03.28 Biogen Idec Adults and children with Hemophilia B


Please Note: “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-3.0] | Wikimedia Commons.

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