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FDA November 2013 Products Receiving Orphan Designation

November 29, 2013

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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Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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