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Orphan Drugs: FDA 2013 In Review

December 31, 2013

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

This Blog Post will review for 2013, the FDA Orphan Drug Designation (ODD) activity. A comparison between the FDA ODD activity for 2012 and 2013 is presented. The source of the information is from the FDA’s Orphan Drug Product Designation Database Application.

I – Comparison of ODDs Granted In 2012 And 2013

The first step in reviewing 2013 is to review the number of FDA ODDs granted in 2013 with the number of FDA ODDs granted in 2012. The chart below compares the numbers for the last two years.

# of 2012 FDA ODDs Granted

# of 2013 FDA ODDs Granted

%   Change From 2012 To 2013

190

257

35.3 %

.

Thus, there is approximately a 35.3 % increase in the number of FDA ODDs granted in 2013 compared to the number of FDA ODDs granted in 2012.

Some of the ODDs granted in 2013 include:

•   Zafgen’s Beloranib for Prader-Willi Syndrome

•   Celgene’s Apremilast for Behcet’s Disease

•   Green Cross Corporation’s (Korea Republic) Idursulfase Beta for Hunter Syndrome

•   Lysogene’s Gene Therapy for Sanfilippo Type A Syndrome

•   Prosensa Therapeutics’ Exon 45/52/53/55 Specific Phosphorothiate Oligonucleotide for Duchenne Muscular Dystrophy

•   National Institutes of Health’s (NIH) Kleptose for Niemann-Pick Disease Type C.

II – Comparison of ODD Approvals In 2012 And 2013

The number of FDA ODDs receiving approval in 2013 is compared to the number of FDA ODDs receiving approval in 2012. The chart below compares the numbers for the last two years.

# of FDA ODDs Receiving Approval In 2012

# of FDA ODDs Receiving Approval In 2013

%   Change From 2012 To 2013

25

30

20 %

.

Thus, there is approximately a 20 % increase in the number of FDA ODDs receiving approval in 2013 compared to the number of FDA ODDs receiving approval in 2012. In 2012, of the 25 ODDs receiving FDA approval, 36% are for an oncology indication. In 2013, of the 30 ODDs receiving FDA approval, 35 % are for an oncology indication.

Some of the ODDs receiving approval in 2013 include:

•   Genzyme’s Kynamro (Mipomersen) for Homozygous Familial Hypercholesterolemia (HoFH)

•   Celgene’s Pomalyst (Pomalidomide) for Multiple Myeloma (MM)

•   Raptor Therapeutics’ Procysbi (Cysteamine Enteric) for Nephropathic Cystinosis

•   Genetech’s Gazyva (Obinutuzumab) for Chronic Lymphocytic Leukemia (CLL)

•   Novo Nordisk’s Tretten (Coagulation Factor XIII A-Subunit Recombinant) Congenital Factor XIII A-Subunit Deficiency.

Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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

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