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FDA February 2014 Products Receiving Orphan Designation

March 2, 2014

The chart below identifies FDA February 2014 Products Receiving Orphan Designation as of 03/02/14 in ascending “Designation Date” order.

Some Observations

•   21 products receive orphan designation in February 2014 (as of 03/02/14)

•   2 products receive orphan designation for Amyotrophic Lateral Sclerosis (ALS)

•  AstraZeneca’s Crestor receives orphan designation for pediatric Homozygous Familial Hypercholesterolemia

•  2 products receive orphan designation for Acute Myeloid Leukemia

•  bluebird bio receives orphan designation for treatment of Sickle Cell Disease.


    Generic Name/ODD Date Sponsor Company Indication
1 Gemcabene/   02.06 Michigan   Life   Therapeutics Homozygous   Familial   Hypercholesterolemia
2 Re188   P2045   somatostatin analog/02.06 Andarix     Pharmaceuticals Small   Cell Lung Cancer
3 T   cell receptor   (TCR) peptide (BV5S2, BV6S5, BV13S1) vaccine/02.06 Immune   Response   BioPharma Pediatric   Multiple   Sclerosis
4 DNA   plasmid vector   (pCK-HGFX7) expressing human hepatocyte growth   factor/02.06 ViroMed   Co Amyotrophic   Lateral   Sclerosis
5 Engineered   variant   of recombinant human fibroblast growth factor 19/02.06 NGM     Biopharmaceuticals Primary   Biliary Cirrhosis
6 Bruton’s tyrosine kinase inhibitor/ 02.10 Celgene Chronic Lymphocytic Leukemia
7 Sotalol   Hydrochloride/02.10 Arbor     Pharmaceuticals VentricularArrhythmiasin Pediatric Patients
8 caprine     hyperimmune serum against HIV lysate/02.14 Daval     International Limited Systemic   Sclerosis
9 [5-(5-Chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine     hydrochloride salt/02.14 Plexxikon Pigmented   Villonodular   Synovitis/Giant CellTumor   of the Tendon


10 Doxofylline/02.14 Alitair     Pharmaceuticals Bronchiectasis
11 Phoxilium/02.14 Gambro   Renal   Products Replacement   Solutionin Patients Undergoing Continuous Renal   Replacement Therapy
12 Bisantrene   HCl/02.14 Update   Pharma Acute   Myeloid Leukemia
13 REMUNE   HIV 1/02.14 Immune   Response   BioPharma Pediatric   HIV/AIDS(age through 16 years)
14 Rosuvastatin     (Crestor)/02.14 AstraZeneca Pediatric   HhomozygousFamilial


15 Gevokizumab/02.21 XOMA PyodermaGangrenosum
16 (6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl]     amino-L-glyceroB-L-manno-heptopyranosyl]amino-9H-purine)/02.21 DARA   BioSciences Chemotherapy-Induced     Peripheral neuropathythat is Refractory to Conventional


17 Autologous   CD34+   hematopoietic stem cells transduced with LentiGlobin BB305   lentiviral vector   encoding the human BA-T87Q-globin gene/02.26 bluebird   bio Sickle   CellDisease
18 Cannabidiol/02.27 GW   Pharma Lennox-GastautSyndrome
19 oligopeptide     containing 6 amino acids (H-Phe-Ser-Arg-Tyr-Ala-Arg-OH)/02.27 Genervon     Biopharmaceuticals AmyotrophicLateral


20 Long   Acting Recombinant   Factor VIIa-CTP3/02.27 PROLOR   Biotech Prophylaxis   of   bleeding episodes in patients with hemophilia A or B with   inhibitors tofactor VIII or factor IX
21 Pracinostat/02.27 MEI   Pharma Acute   Myeloid Leukemia


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

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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