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Orphan Drugs And Rare Diseases: January – February 2013 In Review

April 28, 2013

Note: By popular request, the Orphan Druganaut Blog is republishing this Blog Post. At the beginning of May 2013 (next week), the Orphan Druganaut Blog will publish “Orphan Drugs And Rare Diseases: Review of March – April of 2013”

January thru February 2013 continues the celebration and success for orphan drug development and rare diseases that is achieved in 2012.

A new FDA Breakthrough Therapy Designation is created to further expedite the drug approval process. Section 902, of the July 2012, Food and Drug Administration Safety and Innovation Act (FDASIA), signed by President Obama, defines the new Breakthrough Therapy Designation. In January 2013 Vertex Pharmaceuticals announces that the company is awarded the 1st two FDA Breakthrough Therapy Designations for orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF). Less than 1 month later, on February 12, 2013, Pharmacyclics announces that the company is awarded the 1st two FDA Breakthrough Therapy Designations for oncology – orphan drug Ibrutinib.

Several US FDA regulatory stories for orphan drugs make the headlines :

1) FDA approval of Hyperion Therapeutics’ orphan drug Ravicti (Glycerol Phenylbutyrate) for Urea Cycle Disorders (UCDs)

2) FDA approval of Genzyme and Isis Pharmaceuticals’ orphan drug Kynamro (Mipomersen) for Homozygous Familial Hypercholesterolemia (H0FH)

3) FDA approval of Celgene’s orphan drug Pomalyst (Pomalidomide) for Multiple Myeloma (MM)

4) FDA approval of Bayer and Onyx Pharmaceuticals’ orphan drug Stivarga (Regorafenib) for Gastrointestinal Stromal Tumors (GIST)

5) FDA Orphan Drug Designations (ODDs) given to :

23 products in January 2013 including indications for Duchenne Muscular Dystrophy (DMD), Behcet’s Disease, Prader-Willi Syndrome, and several types of cancers

– 5 products in February 2013 including indications for Niemann-Pick Disease Type C, Hunter Syndrome, and cancers.

6) In January 2013, Pharmaxis receives an unanimous negative recommendation from the FDA Pulmonary-Allergy Drugs Advisory Committee (PADAC) Meeting for orphan drug Bronchitol (Mannitol) for the treatment of Cystic Fibrosis (CF).

Several European (EMA) regulatory stories for orphan drugs make the headlines :

1) EMA Committee for Orphan Medicinal Products’ (COMP) January 2013 Meeting gives 8 positive opinions recommending orphan designation

2) EMA COMP February 2013 Meeting gives 12 positive opinions recommending orphan designation

3) Changes in rates of EMA’s fees for orphan drugs effective January 1, 2013

4) Well publicized campaign to get regulatory approval of Vertex Pharmaceticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) in the UK :

a) NHS in England provides funding starting January 1, 2013

b) SMC in Scotland doesn’t recommend the drug but creates a £21 Million Rare Conditions Medicines Fund, available from March 1, 2013 – April 2014, which will cover the cost of Kalydeco for qualified CF patients

c) Health Service Executive (HSE) in Ireland announces in February 2013, that it will fund Kalydeco

d) Awaiting the Health Technology Appraisal (HTA) in Wales.

Orphan drug companies participate in several important meetings :

  1. J.P. Morgan Healthcare Conference
  2. 15th Annual BIO CEO & Investor Conference
  3. Lysosomal Disease Network WORLD Symposium.

The continuation of the trend from 2012 carries over into 2013 : orphan drugs are the new strategy for Pharma. But this trend is not without controversy as the issue of the high price of orphan drugs is debated by pharma, insurance companies, international regulatory agencies, and in the media.

Some interesting trends are reviewed by the Orphan Druganaut Blog :

  1. Multiple orphan drug indications as part of a company’s life cycle management strategy
  2. Small pharma marketing team of the year for Alexion Pharmaceuticals’ orphan drug Soliris
  3. Launch of new cystic fibrosis organization and website, CFunite
  4. Continuous role of patient advocacy groups on orphan drug development
  5. Big Pharma’s new model of orphan drugs verses the traditional blockbuster drug model
  6. Top rare disease product areas for 2012
  7. Orphan drugs and cancer by the numbers
  8. Reaching out to rare disease patients: smartphone apps
  9. Patient to patient communication with rare disease blogs.

The first two months of 2013 ends a productive and uplifting period for orphan drugs and rare diseases with the global celebration of the 6th Annual International Rare Disease Day 2013.

Please Note: “A group of three balloons on ribbons” JPEG by AJ (Open clip Art Library image’s page) [see page for license] | Wikimedia Commons.

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

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