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What Is The Historic Relationship Between Vertex Pharmaceuticals And FDA’s New Breakthrough Therapy Designation ?

January 6, 2013

       Per a January 6, 2013 Press Release, Vertex Pharmaceuticals announces the 1st two Breakthrough Therapy Designations from the US FDA. 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 of VX-809 with Kalydeco (Ivacaftor) for CF.

The FDA Safety & Innovation Act (S. 3187) or FDASIA is “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.

       The new FDA Breakthrough Therapy Designation is for the purpose of speeding up the development and review of new drugs. In order for a drug to be designated as a Breakthrough Therapy, the sponsor must provide, per Section 902 of FDASIA :

(1) Evidence that the drug is intended , alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition, and

(2) Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

An excellent 20-page document in the Pharmaceutical Law & Industry Report (Vol. 10, NO. 29) titled, “A Guide to the Food and Drug Administration Safety and Innovation Act”, is a great guide to its implementations and impacts on rare diseases, orphan drugs, and the new FDA Breakthrough Therapy Designation.

Another excellent resource is the very detailed 52-page document from the Law Firm, Arnold & Porter LLP, titled,“ A Reference Guide to the Food & Drug Administration Safety and Innovation Act”.


Orphan Druganaut Blog Post, “FDASIA: The Importance of and Impact on Orphan Drug Development”

Orphan Druganaut Blog Post, “Sarepta Therapeutics’ Orphan Drug Eteplirsen and FDASIA”

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

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