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FDA October 2013 Products Receiving Orphan Designation

November 3, 2013

The chart below identifies the 28 FDA October 2013 Products Receiving Orphan Designation. The chart is generated from the FDA’s Orphan Drug Product Designation Database Application and is current as of November 3, 2013. The chart is created by performing a search for the following date range:

Start Date: 10/01/2013 & End Date: 10/31/2013.

Some Observations

•   Stem Cell Therapeutics, a Canadian immuno-oncology company developing cancer stem cell-related therapeutics, receives orphan designation for Tigecycline (FDA-approved antibiotic) for treatment of Acute Myeloid Leukemia (AML)

•   Neurolixis, a San-Diego-based company, receives orphan designation for the treatment of the rare disease, Rett Syndrome

•   Neuren Pharmaceuticals (New Zealand) receives orphan designation for the treatment of the rare disease, Fragile X Syndrome

•   Pharmacyclics receives its 6th and 7th oncology orphan designations for Ibrutinib

•   Gilead Sciences receives its 3rd – 6th oncology orphan designations for Idelalisib

•   50% of the orphan indications are for an oncology indication.

Row Num

Generic Name

Designation Date

Orphan   Designation

1

                                                                      3-chloro-4-fluorophenyl-[4-fluoro-4-[[(5-methylpyrimidin-2-ylmethyl)amino]methyl]piperidin-1yl]methanone 10-25-2013  Rett Syndrome

2

      6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide 10-15-2013 Stage IIb-IV Melanoma

3

      Gallium-68   (DOTA0-Phel-Tyr3)octreotide 10-23-2013 Neuroendocrine Tumors

4

      PEGylated   recombinant anti-Pseudomonas aeruginosa PcrV Fab’ antibody 10-25-2013 Cystic Fibrosis with Pseudomonas Aeruginosa Lung Infection

5

      TXA127 10-25-2013 Acute Radiation Syndrome

6

      Flubendazole 10-25-2013 Onchocerciasis

7

Fusion protein analog with recombinant human   growth hormone (rhGH) at once-a-month dosing 10-16-2013 Growth Hormone Deficiency

8

      Glycyl-L-2-methylprolyl-L-glutamic   Acid 10-23-2013 Fragile X Syndrome

9

      Human   IgG1k monoclonal antibody 10-16-2013 Systemic Sclerosis

10

      Human   monoclonal antibody againt human interleukin 13 (IL-13) 10-15-2013 Eosinophilic Esophagitis

11

      Ibrutinib 10-23-2013 Diffuse Large B-Cell Lymphoma

12

      Ibrutinib 10-15-2013 Waldenstrom’s macroglobulinemia

13

      Idelalisib 10-15-2013 Nodal Marginal Zone Lymphoma

14

Idelalisib 10-15-2013 Chronic Lymphocytic Leukemia & Small Lymphocytic Lymphoma

15

      Idelalisib 10-15-2013 Extranodal Marginal Zone Lymphoma

16

      Idelalisib 10-15-2013 Splenic Marginal Zone Lymphoma

17

      Isavuconazonium   sulfate 10-25-2013 Zygomycosis

18

      mTOR   kinase inhibitor (CC-223) 10-16-2013 Hepatocellular Carcinoma

19

      Olaparib 10-16-2013 Ovarian Cancer

20

      Oleylphosphocholine 10-25-2013 Leishmaniasis

21

      Phenobarbitol   sodium injection 10-23-2013 Hypoxic-Ischemic Encephalopathy

22

      Poly(lactide-co-glycolide)   carboxylated microparticle 10-25-2013 Acute Encephalitis Syndrome

23

     Recombinant   human monoclonal IgM antibody targeting glucose regulated protein 78 10-25-2013 Multiple Myeloma

24

      Sodium   fusidate 10-23-2013 Prosthetic Joint Infections

25

      Tigecycline 10-23-2013 Acute Myeloid Leukemia.

26

      Tivantinib 10-16-2013 Hepatocelular Carcinoma

27

      Trastuzumab   emtansine 10-25-2013 Gastric Cancer

28

      Trehalose 10-25-2013 Occulopharyngeal Muscular Dystrophy

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

Please Note: “DNA Purification” Courtesy of Dr. Bruce Chassey Laboratory. National Institute Of Dental Research. Li-shan. NCI [Public domain in the US].

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

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