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FDA Breakthrough Therapy Designation Statistics

March 1, 2013

The FDA’s Chief Scientist, Jesse L. Goodman, MD, MPH, gives a presentation on February 27, 2013, to the FDA Science Board and discusses the status of the FDA’s new Breakthrough Therapy Designation :

  1. 22 Breakthrough Therapy Designation requests are received
  2.   5 Breakthrough Therapy Designation requests are granted
  3.   5 Breakthrough Therapy Designation requests are denied
  4. 12 Breakthrough Therapy Designation requests are pending.

Of the 5 Breakthrough Therapy Designations granted :

  1. Two are awarded to Vertex Pharmaceuticals for expanding the uses of its Cystic Fibrosis drug Kalydeco
  2. Two are awarded to Pharmacyclics and Janssen Research & Development for the cancer therapy drug Ibrutinib
  3. The fifth and last designation is unknown at this point in time.

An interesting article from Elsevier Business Intelligence, “Vertex Phase III For CF Combo Shows Power of Breakthrough Designation” (may need subscription for access), discusses the new announcement from Vertex Pharmaceuticals this past week that the pivotal Phase III clinical trials for the combination of VX-809 + Kalydeco is to measure efficacy and safety over 24 weeks. On the other hand, for Kalydeco monotherapy, the FDA approves the drug in 2012 based on 56 weeks of clinical trial data. Thus, the “power of the Breakthrough Therapy Designation”.

The FDA has a Breakthrough Therapy Designation “Frequently Asked Questions” section on their website.  Here are a few highlights :

  1. A FDA guidance on the Breakthrough Therapy Designation is currently in development. The FDA will issue a draft guidance by January 2014 – no later than 18 months after the implementation of FDASIA, which is signed into law by President Obama in July 2012.
  2. A Breakthrough Therapy Designation request can be submitted for each of the multiple indications for the same drug
  3. The FDA will not disclose information regarding sponsors who submit requests for, or who have been granted or denied Breakthrough Therapy Designations
  4. All the benefits of a Fast Track Designation are available to drugs that have received a Breakthrough Therapy Designation.

To expedite the development and review of drugs for life-threatening and serious diseases, the FDA had three programs before the Breakthrough Therapy Designation is created :

– Fast Track

– Accelerated Approval

– Priority Review.

The three Designations are in existence for at least 20 years. What is different about the Breakthrough Therapy Designation is the “ … requirement of early clinical data demonstrating an unprecedented effect …”. For example, the Fast Track Designation can be granted off the back of promising preclinical data, whereas the Accelerated Approval Designation is about surrogate trial endpoints. For a Breakthrough Therapy Designation, the FDA is expected to work closely with drug sponsors to “ …. design collaborative, multidisciplinary development plans that hasten timelines to approval and minimize the number of patients exposed to less efficacious treatment or placebos ….”.

References February 27, 2013 article titled, “The Vitals: FDA receives additional breakthrough designation requests”  (may need subscription or trial subscription for access).

Please Note: “Idea” PNG by Producer at ar.wikipedia (Transferred from ar.wikipedia) [Public domain] | Wikimedia Commons.

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

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