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Orphan Drugs And The New FDA Breakthrough Therapy Designation

January 23, 2013

To expedite development and review of drugs for life-threatening and serious diseases, the FDA had 3 programs :

–  Fast Track

–  Accelerated Approval

–  Priority Review.

A new FDA Breakthrough Therapy Designation has recently been created to further expedite the drug approval process.  Section 902, of the July 9, 2012, Food and Drug Administration Safety and Innovation Act (FDASIA), signed by President Obama, defines the new Breakthrough Therapy Designation. In order for a drug to be designated as a Breakthrough Therapy, the sponsor must provide :

(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 introduction to the new Breakthrough Therapy, is a video from titled, “Urgency”, where the following 2 people discuss the history, purpose, and review of this new drug approval pathway :

–   Dr. Jane Woodchuck, Director of FDA’s Center for Drug Evaluation and Research, who will oversee the implementation

–   Jeff Allen, PhD, Executive Director of Friends of Cancer Research, the advocacy group proposed the new model, the Breakthrough Therapy drug pathway.

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 orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF).  Vertex Pharmaceuticals’ 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.


FDA Fact Sheet: Breakthrough Therapies 

*** Friends of Cancer Research article titled, “RPM Report – Breakthrough Therapy: New Pathway in FDASIA May Point The Way To Future Reforms”

FDA Pharmaceutical Law & Industry Report (2012,Vol. 10, NO. 29), “A Guide to the Food and Drug Administration Safety and Innovation Act”

Arnold & Porter LLP, 2012, “ A Reference Guide to the Food & Drug Administration Safety and Innovation Act”.

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

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