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What Orphan Drug Receives The 1st FDA New Breakthrough Therapy Designation ?

January 8, 2013

Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis receives the 1st 2 US FDA Breakthrough Therapy Designations in FDA’s history.

The FDA for the past few months would not identify the company or product that had received the 1st FDA Breakthrough Therapy Designation. It is in December 2012, that Janet Woodcock, Director of FDA’s Center for Drug Evaluation and Research, in the FDA’s December 3, 2012, Voice Blog Post, identifies that the agency accepted the 1st “breakthrough product designation” submissions.  But the FDA refused to identify which company or companies owned the submissions, and also refused to identify the product.

Per a January 6, 2013 Press Release, Vertex Pharmaceuticals announces that the company is awarded the 1st two Breakthrough Therapy Designations from the US FDA for their orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF). 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 :

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

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


FDA Voice Blog December 3, 2012 Post titled, “Advancing ‘Breakthrough’ Drug Therapies” January 8, 2013 article titled, “A Conversation With a Breakthrough Drug”

Orphan Druganaut Blog Posting titled, “What is the Historic Relationship Between Vertex Pharmaceuticals and FDA’s New Breakthrough Therapy Designation ?”

Regulatory Focus (RF) Organization December 4, 2012 article titled, “Woodcock Lauds First Submission of ‘Breakthrough’ Product”

Regulatory Focus (RF) Organization January 8, 2013 article titled, “Solved: Owner of 1st 2 ‘Breakthrough Product Designations” Revealed”

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

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