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FDA December 2013 Products Receiving Orphan Designation

December 29, 2013

Please note: Updated 01/02/14 with 12/31/13 FDA Updates.

The chart below identifies FDA December 2013 Products Receiving Orphan Designation as of 01/02/14 in ascending “Designation Date” order.

Some Observations

•   21 products receive orphan designation in December 2013 (as of 01/02/14)

•   2 products receive orphan designation for Multiple Myeloma

•   Canakinumab receives orphan designation for 2 indications

•   20 % of the orphan designations are for an oncology indication.

FDA December 2013 Products Receiving Orphan Designation

Num

Generic Name Designation Date Orphan Designation

1

                                                                      Canakinumab 12-05-2013 Familial Mediterranean Fever

2

     Canakinumab 12-05-2013 Hyperimmunoglobulinemia D

and

Periodic Fever Syndrome

3

     Sotatercept 12-05-2013 Beta-Thalassemia Intermedia

and Major

4

     O2-(2,4-dinitrophenyl)   1-[(4-ethoxycarbonyl)piperazin-1yl]diazen-1-ium-1,2-diolate 12-13-2013 Multiple Myeloma

5

     Marizomib   12-13-2013 Multiple Myeloma

6

     Human   monoclonal antibody inhibitor of mannan-binding lectin-associated serine   protease-2 (MASP-2) 12-16-2013 Prevention (inhibition) of Complement-Mediated Thrombotic Microangiopathy

7

     Metadoxine 12-16-2013 Fragile X Syndrome

8

     Onartuzumab   (MetMab) 12-16-2013 Hepatocellular

Carcinoma

9

Vintafolide 12-16-2013 Ovarian Cancer

10

     CD40/CD80/CD86   modified autologous dendritic cell therapy 12-20-2013 Type 1 Diabetes MellitusWith residual beta cell function

11

     Tacrolimus 12-20-2013 Prophylaxis of organ rejection For allogenheic kidney   transplant

12

Erdosteine 12-20-2013 Bronchiectasis

13

     Fenfluramine   HCI 12-20-2013 Dravet Syndrome

14

     Tolcapone 12-24-2013 Transthyretin Amyloidosis

15

     Recombinant   Human Acid Ceramidas 12-24-2013 Farber Disease

16

                                                               N-acetyl   cysteine amide 12-31-2013 Retinitis Pigmentosa

17

     NorLeu3-Angiontensin(1-7))   [NorLeu3-A(1-7)] 12-31-2013 Leakage from surgical site following penetrating   keratoplasty

18

     Gallium   [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide 12-31-2013 Diagnostic for the Management of Gastro-Entero-Pancreatic   Neuroendocrine Tumors

19

     thymosin   beta 4 12-31-2013 Neurotrophic Keratopathy

20

     nifurtimox 12-31-2013 Chagas Disease

21

     radiolabeled   somastatin analog 12-31-2013 Diagnostic for the management of neuroendocrine tumors

.

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

FDA December 2013 Orphan Designations Receiving Approval

The chart below identifies the Orphan Designations receiving FDA Decemberer 2013 approval  as of 01/02/14.

 

Row Num

Generic Name

Marketing   Approval Date

Approved Labeled   Indication

1

                                                                      collagenase   clostridium histolyticum 12-06-2013 Peyronie’s Disease

2

Anti-Inhibitor Coagulant Complex 12-16-2013 Routine prophylaxis to prevent or bleeding episodes in   hemophilia A/ B

3

Factor XIII [A2} homodimer, recombinant DNA origin 12-23-2013 Prevention  of   bleeding associated with congential FXIII deficiency

.

Please Note: “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-3.0] | Wikimedia Commons.

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

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