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FDA September 2014 Products Receiving Orphan Designation

September 30, 2014





The chart below identifies FDA September 2014 Products Receiving Orphan Designation as of 09/30/14 in ascending “Orphan Drug Designation Date” order.

FDA September 2014 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1 Mebendazole/ 09.03 Janssen Pharmaceutical Single or mixed gastrointestinal infestations by Trichuris trichiura (whipworm), Ascaris lumbricoides (large roundworm), and Ancylostoma duodenale and Nectar americanus (hookworm)
2 Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter/ 09.04 Columbia University Medical Center Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
3 Adenovirus serotype-5 (Ad5) vector that contains a modified non-oncogenic fused early 6 (E6) and early 7 (E7) gene of the human papillomavirus (HPV); (Ad5 [E1-, E2b-]-E6/E7) / 09.04 Etubics Corporation HPV-associated head and neck squamous cell carcinoma (HNSCC)
4 5,7-dichloro-2-methylaminomethyl-8-hydroxyquinolone hydrochloride / 09.04 Prana Biotechnology Limited Huntington’s Disease
5 6-((3S,4S)-4-Methyl-1-pyrimidin-2-ylmethyl-pyrrolidin-3-yl-1-(tetrahydro-pyran-4-yl)-1,5-dihydro-pryazolo[3,4-d]pyrimidin-4-one / 09.04 Pfizer Sickle Cell Disease
6 N-(2-{2-dimethylaminoethyl-methylamino}-4-methoxy-5-{[4-(1-methylindol-3-yl)pyrimidin-2-yl]amino}phenyl)prop-2-enamide mesylate salt/ 09.04 AstraZeneca Epidermal growth factor receptor mutation-positive NSCLC
7 inBreath airway transplant system/ 09.04 Harvard Apparatus Regenerative Technology Restore the structure and/or function of the trachea
8 Ibrutinib / 09.08 Pharmacyclics Follicular lymphoma
9 mitomycin and sterile hydrogel composed of poloxamer 407, hydroxypropyl methyl cellulose and polyethylene glycol 400/ 09.08 TheraCoat (Israel) Upper tract urothelial cell cancers
10 vemurafenib / 09.08 Genentech NSCLC with BRAF V600E mutation
11 polymer surgical mesh (3S)-1-azabicyclo[2.2.2]oct-3-yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate/ 09.11 Genzyme Gaucher Disease
12 Cysteamine/ 09.11 NovaBiotics Ltd. (UK) Cystic Fibrosis
13 humanized monoclonal antibodies hu1B7 and hu11E6/ 09.11 Synthetic Biologics Bordetella pertussis
14 silibinin-C-2′,3-dihydrogensuccinate, disodium salt/ 09.11 Rottapharm S.p.A. (Italy) Amatoxin poisoning
15 technetium Tc 99m tilmanocept / 09.17 Navidea Biopharma Use in sentinel lymph node detection (SLN) For cancer of the head and neck
16 4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl) cyclopent-2-en-1-yl] pyrimidin-2-one/ 09.23 Rexahn Pharmaceutials Pancreatic Cancer
17 MV-NB-02, its bivalent ganglioside vaccine consisting of GD2-lactone and GD3-lactone each covalently conjugated to keyhole hemocyanin/ 09.23 MabVax Therapeutics Neuroblastoma
18 heat killed whole cell mycobacterium obuense/ 09.23 Immodulon Therapeutics Limited (UK) Pancreatic Cancer
19 nitric oxide/ 09.23 Advanced Inhalation Therapies (Israel) Cystic Fibrosis
20 cannabidiol/ 09.24 Insys Therapeutics Glioma
21 aldoxorubicin/ 09.24 CytRx Corporation Glioblastoma multiforme
22 aldoxorubicin/ 09.24 CytRx Corporation Small Cell Lung Cancer
23 aldoxorubicin/ 09.24 CytRx Corporation Ovarian Cancer
24 glucagon infusion/ 09.25 Xeris Pharmaceuticals Prevention of chronic, severe hypoglycemia related to congential hyperinsulinism


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


•   ODDs for Rare Diseases:

1.   Gaucher Disease
2.   Huntington’s Disease
3.   Sickle Cell Disease
4.   Cystic Fibrosis

•   50% of ODDs are for an Oncology Indication

•   ODD for a vaccine for childhood cancer Neuroblastoma

•   ODDs by several Big Pharma companies:

1.   Pfizer
2.   AstraZeneca
3.   Genzyme
4.   Genentech
5.   Janssen Pharmaceutical
6.   Pharmacyclics (Ibrutinib receives its 8th ODD).

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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