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FDA Breakthrough Therapy Designation Statistics (04.08.13)

April 8, 2013

Pharmacyclics and Janssen Research & Development announces on April 8, 2013, that the FDA grants a third oncology Breakthrough Therapy Designation for orphan drug Ibrutinib. The designation is for the use of Ibrutinib as a monotherapy for Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) with deletion of the short arm of chromosome 17. Patients harboring a deletion within chromosome 17 generally have poor response to chemoimmunotherapy and have limited treatment options. The presence of deletion 17p is one of the worst prognostic factors in patients with CLL.

In February 2013, Pharmacyclics announces that the company is awarded the first two oncology Breakthrough Therapy Designations from the US FDA for their orphan drug Ibrutinib :

• Ibrutinib monotherapy for the treatment of patients with relapsed or refractory Mantle Cell Lymphoma (MCL)
• Ibrutinib monotherapy for the treatment of patients with Waldenstrom’s Macroglobulinemia (WM).

The FDA updates on April 8, 2013, the statistics on their website for Breakthrough Therapy Designations, for the period July 9, 2012 – March 31, 2013 (CDER + CBER) :

•   40 Breakthrough Therapy Designation requests are received
•   10 Breakthrough Therapy Designation requests are granted
•   12 Breakthrough Therapy Designation requests are denied
•   18 Breakthrough Therapy Designation requests are pending.

Of the 10 Breakthrough Therapy Designations granted :

•  Two are awarded to Vertex Pharmaceuticals for expanding the uses of its Cystic Fibrosis orphan drug Kalydeco
•  Three are awarded to Pharmacyclics and Janssen Research & Development for the cancer therapy orphan drug Ibrutinib
•  One is awarded to Novartis for cancer non-orphan drug LDK378
•  The remaining 4 designations are unknown at this point in time.

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

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 The FDA will issue a draft guidance by January 2014 – no later than 18 months after the implementation of FDASIA, which is signed into law by President Obama in July 2012.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

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. 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 ….”.

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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