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FDA January 2015 Products Receiving Orphan Designation

January 30, 2015





The chart below identifies FDA January 2015 Products Receiving Orphan Designation as of 01/30/15 in ascending “Orphan Drug Designation Date” order.

FDA January 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1  amikacin sulfate/ 01.05 PlumeStars s.r.l. (Italy) cystic fibrosis with Pseudomonas aeruginosa lung infections
2 celiprolol/ 01.05 Acer Therapeutics Ehlers-Danlos syndrome
3 Sparsentan/ 01.05 Retrophin LLC Focal Segmental Glomerulosclerosis
4 Pyrvinium/ 01.05 StemSynergy Therapeutics familial adenomatous polyposis
5 Pentosan polysulfate sodium/ 01.05 Plexcera Therapeutics MPS VI
6 Selinexor/ 01.05 Karyopharm Therapeutics Multiple Myeloma
7 Simtuzumab/ 01.05 Gilead Sciences primary sclerosing cholangitis
8 monoclonal antibody targeting eotaxin-2/ 01.06 ChemomAb Ltd (Israel) Systemic Sclerosis
9 N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt/ 01.07 Atterocor Cushing’s Syndrome
10 Ulocuplumab/ 01.12 Bristol-Myers Squibb acute myeloid leukemia
11 deutetrabenazine/ 01.13 Auspex Pharmaceuticals Tourette Syndrome
12 anti-Beta 1 integrin monoclonal antibody/ 01.13 OncoSynergy Ovarian Cancer
13 arimoclomol citrate/ 01.13 Orphazyme ApS (Denmark) Neimann-Pick disease, type C
14 Naltrexone/ 01.13 TaiwanJ Pharmaceuticals (Taiwan) Autoimmune Hepatitis
15 pentapeptide with sequence Ac-VSRRR-NH2/ 01.13 CereNOva LLC intracerebral hemorrhage
16 2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5-/ 01.15 Neurocrine Biosciences congenital adrenal hyperplasia (CAH)
17 5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamide/ 01.15 Gilead Sciences pulmonary arterial hypertension
18 autologous CD34+ bone marrow derived stem cells transduced with a self-inactivating gammaretroviral vector encoding the human IL2RG (yc) / 01.15 Boston Children’s Hospital severe combined immune deficiency-X1
19 pegylated carboxyhemoglobin bovine/ 01.152 Prolong Pharmaceuticals Sickle Cell Disease
20 synthetic oligomer of 16 nucleotides / 01.15 Isis Pharmaceuticals myotonic dystrophy Type I
21 8-(1-Hydroxy-ethyl)-2-methoxy-3-(4-methoxy-benzyloxy)-benzo[c]chromen-6-one/ 01.21 RestorGenex Corporation glioblastoma multiforme
22 liposomal encapsulated paclitaxel/ 01.21 Insys Therapeutics Ovarian Cancer
23 Antroquinonol/ 01.21 Golden Biotechnology Corp (Taiwan) Pancreatic Cancer
24 recombinant humanized anti-tau antibody/ 01.22 C2N Diagnostics progressive supranuclear palsy
25 (S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile/ 01.26 Almirall S.A. (Spain) Pemphigus Vulgaris
26 18Fluorine-N-3-Fluoropropyl-2beta-carbomethoxy-3beta-(4-iodophenyl) Nortropane/ 01.21 Advanced Imaging Projects Diagnostic to be used in the management of multiple system atrophy (MSA)
27 2-(3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-l-yl)-1-(1-(3-fluoro-2-(trifluoromethyl)isonicotinoyl)piperidin-4-yl)azetidin-3-yl)acetonitrile adipate/ 01.26 Incyte Corp Pancreatic Cancer
28 double stranded RNA which targets the mutated KRAS oncogene/ 01.26 Silenseed Ltd (Israel) Pancreatic Cancer
29 leukotriene A4 hydrolase inhibitor/ 01.26 Celtaxsys Cystic Fibrosis
30 Theranost 68Ga RGD/ 01.26 Advanced Imaging Projects Diagnostic for the clinical management of patients with tuberculosis
31 human leukocyte antigen-A2 restricted peptides/ 01.26 Stemline Therapeutics Glioma
32 Tarextumab/ 01.26 OncoMed Pharmaceuticals small cell lung cancer
33 Tarextumab/ 01.26 OncoMed Pharmaceuticals Pancreatic Cancer
34 lisinopril oral solution/ 01.27 codaDose Primary & Secondary Hypertension
35 Alectinib/ 01.27 Genentech ALK+ NSCLC
36 Filgrastim/ 01.27 Neurovision Pharma GmbH ALS
37 recombinant human fucosyltransferase VI [ FTVI ] + GDP-fucose/ 01.27 TargaZyme prevention of graft versus host disease


** 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-30] | Wikimedia Commons.

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