FDA November 2013 Products Receiving Orphan Designation

Please Note: Blog Post Updated 12/02/13 & 12/03/13 to add FDA Updates.

Some Observations

•   23 products receive FDA Orphan Drug Designation (ODD) in November 2013

•   3 FDA ODDs receive approval in November 2013

•   38.5 % of the orphan indications are for an oncology indication

•   BioProducts Laboratory Limited (UK) receives an orphan indication for the rare disease, Sickle Cell Disease

•   Genzyme is granted an orphan designation for Pompe Disease

•   Gen Sight Biologics (France) gets an orphan designation for a gene therapy for Leber Hereditary Optic Neuropathy

•   Dyax Corporation receives an orphan indication for treatment of Hereditary Angioedema (HAE).

FDA November 2013 Products Receiving Orphan Designation

The chart below identifies the 23 FDA November 2013 Products Receiving Orphan Designation as of 12/03/13.

Row       Num	Generic       Name	Designation       Date	Orphan       Designation
1	2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol	11-14-2013	Dravet     syndrome.
2	5-((4-bromo-2-fluorophenyl)amino)-4-fluoro-N-(2-hydroxyethoxy)-1-methyl-1H-benzo[d]imidazole-6-carboxamide	11-19-2013	Stage     IIB-IV melanoma
3	Angiotensin     (1-7)	11-26-2013	Limb-Girdle     Muscular Dystrophy
4	Filgrastim	11-20-2013	Myelosuppression
5	Lonafarnib	11-19-2013	Hepatitis     Delta Virus   (HDV)infection
6	MEK162   + LGX818	11-19-2013	Stage     IIB-IV melanoma positivefor   the BRAF mutation.
7	Methyl       [(2S)-1-{[4-(3-{5-chloro-2-fluoro-3-[(methylsulfonyl)amino]phenyl}-1-isopropyl-1H-pyrazol-4-yl)-2-pyrimidinyl]amino}-2-propanyl]carbamate	11-19-2013	Stage     IIB-IV melanoma postive forBRAF mutation
8	Pegfilgrastim	11-20-2013	Myelosuppression
9	Poloxamer   188	11-08-2013	Acute     Limb Ischemia
10	Siponimod	11-26-2013	Polymyositis
11	adeno-associated   viral vector containing the human NADH     Dehydrogenase 4 Gene	11-20-2013	Leber     Hereditary Optic Neuropathy
12	autologous   CD4+CD25hiFoxP3+regulatory T cells	11-19-2013	Prevention     of graft rejection
13	bendamustine   hydrochloride	11-26-2013	Different     Types of Lymphomas
14	eltrombopag	11-08-2013	Aplastic     Anemia
15	emricasan	11-20-2013	Liver     transplant recipients with   reestablished fibrosis
16	hRS7-SN-38   IgG Conjugate	11-27-2013	Small   Cell Lung Cancer
17	human   haptoglobin	11-19-2013	Sickle     Cell Disease
18	Human     Monoclonal Antibody Directed Against Active Plasma Kallikrein	11-16-2013	Hereditary     Angioedema (HAE)
19	Human Umbilical Cord Blood-Derived Mesenchymal Stem Cells	11-26-2013	Bronchopulmonary   Dysplasia
20	live   attenuated bioengineered Listeria monocytogenes     immunotherapy	11-04-2013	Human     Papilloma Virus-Associated Head/Neck Cancer
21	monoclonal   antibody directed at hepatitis C virus E2     glycoprotein	11-04-2013	Hepatitis     C recurrence
22	Pirfenidone	11-19-2013	Systemic     Sclerosis
23	recombinant   human alpha-glucosidase conjugated with     synthetic   bis-mannose-6-phosphate-Man6 glycan	11-19-2013	Pompe     Disease

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

FDA November 2013 Orphan Designations Receiving Approval

The chart below identifies the 3 Orphan Designations receiving FDA November 2013 approval as of 12/03/13.

Row     Num	Generic     Name	Designation     Date	Orphan     Designation	Marketing     Approval Date	Approved     Labeled Indication
1	obinutuzumab	02-17-2012	Treatment   of chronic lymphocytic   leukemia	11-01-2013	Chronic   Lymphocytic Leukemia
2	ibrutinib	12-03-2012	Treatment   of mantle cell lymphoma	11-13-2013	Mantle   cell lymphoma (MCL)
3	Sorafenib	12-12-2011	Thyroid   Cancers	11-22-2013	Thyroid   Cancer

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

Please Note: “Petri Dish” courtesy of Linda Bartlett (Photographer). National Institute Of Health [Public domain] | Wikimedia Commons.

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