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Orphan Drugs And Rare Diseases: Review of 2012

January 2, 2013

      2012 has been an interesting year for orphan drugs and rare diseases.

The topic of a new model of orphan drugs for Big Pharma and its profitability is the main Internet Buzz during the Summer of 2012, when Thomson Reuters comes out with their article titled, “The New Blockbusters: Orphan Drugs”. Big Pharma’s new strategic model of orphan drugs and rare diseases starts to replace Big Pharma’s traditional blockbuster drug model. Prof. M. Ian Phillips, Director of the Center for Rare Disease Therapies at the Keck Graduate Institute of Applied Life Sciences in California, writes an Editorial in the  January 2013 issue of Expert Opinion on Orphan Drugs on this topic.

A historic regulatory event in Canada occurs when Canada’s Health Minister Aglukkaq announces the “1st ever Canadian framework to increase access to new treatments and information & the launch of Orphanet-Canada”.

Another news story in 2012 that creates Internet buzz, is the death of US actor Jack Klugman and the little known story about his role in helping to pass the US Orphan Drug Act through Congress in 1983.

The FDA Safety & Innovation Act (S. 3187) or FDASIA is enacted after President Obama signs it on July 9, 2012.  FDASIA “is a big step towards the development of effective and safe treatments for rare diseases and orphan drug development in the United States.” This legislation is to have an important and large impact on the future of orphan drug development :

1) Implementation of a rare pediatric disease priority review voucher incentive program

2) New “breakthrough therapy” category for drug approval

3) Consultation with rare disease medical experts

4) Acceleration of new medical treatments for patient access.

Legal battles are a key part of orphan drug activity in 2012, especially when it comes to the topic of “orphan drug designation” and “orphan drug exclusivity” :

1) K-V Pharmaceutical’s orphan drug Makena, resulting in the company taking legal action against the FDA and several US State Medicaid Departments

2) FDA’s decision to “rescind” Octapharma’s orphan drug exclusivity for Wilate

3) Depomed filing a complaint questioning why the FDA gives orphan drug Gralise an “orphan drug designation”, but not “orphan drug exclusivity”.

Innovative gene therapy, which has the “potential to cure lethal diseases by enabling normal genes to take over for defective ones” gains momentum in 2012 :

1) Orphan designation by both the FDA and European Medicines Agency (EMA) for Bluebird Bio’s gene therapy product for Adrenoleukodystrophy (ALD)

2) European Union (EU) approval of orphan designated Glybera – the “1st gene-therapy medicine approval in the Western world”, for the treatment of a lipoprotein lipase deficiency.

Several regulatory stories of orphan drugs make the headlines in 2012 :

1) EMA rejects Pfizer and Protalix BioTherapeutics’ Elelyso for Gaucher Disease

2) EMA’s Medicinal Products for Human Use (CHMP)  issues negative recommendation for Isis Pharmaceuticals’ Marketing Authorization Application (MAA) for Kynamro (Mipomersen) on December 13, 2012

3) FDA approval of NPS Pharmaceuticals’ orphan drug Gattex (Teduglutide) for Short Bowel Syndrome

4) FDA approval of Exelisis’ orphan drug Cometriq (Cabozantinib) for Thyroid Cancer

5) Personalized medicine – FDA, EMA, and Canadian approval of Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for a specific Cystic Fibrosis (CF) gene mutation

6) FDA approval of Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide) for Homozygous Familial Hypercholesterolemia (HoFH).

In the United States in the 2012 calendar year :

1) There are 188 FDA orphan drug product designations compared to 203 in 2011, 194 in 2010, and 164 in 2009.

According to a December 2012 FDA Report, for the FDA 2012 Fiscal Year (FY) (October 1, 2011 – September 30, 2012) :

1) FDA approves 35 New Molecular Entities (NMEs) in its fiscal year 2012

2) Of the 35 NMEs for 2012, 9 or approximately 26% are for orphan drugs

3) In the last 5 years, approximately 33% of the NMEs are for rare diseases

4) 7 of the 9 or approximately 78% of the orphan drugs are “approved in the 1st cycle”

5) 8 of the 9 or approximately 89% of the orphan drugs had their PDUFA dates met

6) Of the 9 or approximately 67% of the orphan drugs were 1st approved in the US.

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

  1. Reblogged this on Russell-Silver Syndrome and commented:
    Facts and Stats: 2012 Orphan Drug and Rare Disease Recap

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