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What Awards And Firsts Has Vertex Pharmaceuticals’ Orphan Drug Kalydeco Achieved ?

January 21, 2013

Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) has won several awards and recognitions :

  1)  The Wall Street Journal’s 2012 Technology Innovation Award For Medicine and Biotech

The Wall Street Journal received 536 applications from more than 24 countries. Wall Street Journal reporters and editors reviewed all entries and 172 were then forwarded to an independent panel of judges. From that group, judges chose a total of 37 winners and runners-up in 18 categories.

2)   Forbes’s Most Important New Drug Of 2012

      Matthew Herper, Forbes’ Pharma & Healthcare writer, calls Kalydeco the most important new drug of 2012 for 3 reasons :

–   Triumph of Personalized Medicine

–   Power of a patient advocacy organization, the Cystic Fibrosis Foundation, for helping in the development of the drug

–   Price : $294,000/year/patient.

3)  Receives The 1st 2 FDA New Breakthrough Therapy Designations

Per a January 6, 2013 Press Release, Vertex Pharmaceuticals announces that the company is awarded the 1st two FDA Breakthrough Therapy Designations for Kalydeco. Under Section 902 of the July 9, 2012, Food and Drug Administration Safety and Innovation Act (FDASIA) signed by President Obama, the new FDA Breakthrough Therapy Designation is described.

Vertex Pharmaceuticals identifies the 2 FDA Breakthrough Therapy Designations :

–  FDA’s 1st Breakthrough Therapy Designation is granted to Kalydeco Monotherapy for CF for potential additional indications beyond the current approval for CF patients 6 & older with the G551D mutation

–  FDA’s 2nd Breakthrough Therapy Designation is granted for the combination therapy of VX-809 with Kalydeco for CF.

References January 8, 2013 article titled, “A Conversation With a Breakthrough Drug”

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

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