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FDA September 2013 Products Receiving Orphan Designation (Part 1)

October 1, 2013

There are 42 products receiving FDA Orphan Designation for September 2013 – the most of any month for 2013. Because of the large number of Orphan Designations, the list is split into 2 charts in 2 Blog Posts.

The chart below identifies the FDA September 2013 Products Receiving Orphan Designation. The chart is generated from the FDA’s Orphan Drug Product Designation Database Application and is current as of October 1, 2013. The chart is created by performing a search for the following date range:

Start Date: 09/01/2013 & End Date: 09/31/2013.

Row Num

Generic Name

Designation Date

Orphan   Designation

1

                                                                      (4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane   Chloride 09-04-2013 Primary Biliary Cirrhosis

2

     (4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane   Chloride 09-04-2013 Primary Sclerosing Cholangitis

3

     (4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane   Chloride 09-04-2013 Progressive Familial Intrahepatic Cholestasis

4

     (4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane   Chloride 09-04-2013 Alagille Syndrome

5

     1-(4-benzhydrylpiperazin-1-yl)-3,3-diphenylpropan-1-one 09-26-2013 Postherpetic Neuralgia

6

     4-[(5R)-6,7-Dihydro-5H-pyrrolo[1,2-c]imidazol-5-yl]-3-flurorbenzonitrile 09-13-2013 Cushing’s Disease

7

     6,8-bis-benzylsulfanyl-octanoic   acid 09-26-2013 Myelodysplastic Syndrome

8

     L-arginyl-L-isoleucyl-L-valyl-L-prolyl-L-alanine-amide 09-11-2013 Acute Radiation Syndrome

9

     LDK378 09-27-2013 ALK+ Non-Small Cell Lung Cancer (NSCLC)

10

     Nitric   oxide 09-11-2013 Cystic Fibrosis

11

     OMS643762 09-26-2013 Huntington’s Disease

12

     aadeno-associated   viral vector, serotype 2, containing the human choroideremia gene encoding   human Rab escort protein 1 09-12-2013 Choroideremia

13

     anti-CD3   mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A   chain fusion protein 09-13-2013 Graft versus Host Disease

14

     anti-sense   oligonucleotide consisting of 2’0-Me RNA with a phosphorothioate backbone 09-23-2013 Cystic Fibrosis

15

     autologous   ex vivo expanded CD4+-enriched leukocytes treated with the de-methylating   agent 5-aza-2’-deoxycytidine 09-13-2013 Glioblastoma Multiforme

16

     bacterium   Bacteroides thetaiotaomicron 09-26-2013 Active Crohn’s Disease in Pediatrics

17

     bendamustine   hydrochloride with betadex sulfobutyl ether sodium 09-10-2013 Chronic Lymphocytic Leukemia (CLL)

18

     brentuximab   vedotin 09-13-2013 Angioimmunoblastic T-cell Lymphoma

19

     chimeric   monoclonal antibody against claudin-18 splice variant 2 09-26-2013 Pancreatic Cancer

20

     cysteamine 09-11-2013 Pancreatic Cancer

21

     dehydrated   alcohol 09-11-2013 Hypertrophic Obstructive Cardiomyopathy

**  “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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