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Orphan Drugs And New FDA Breakthrough Therapy Regulatory Pathway: An Update

February 12, 2013

The new FDA breakthrough therapy regulatory pathway started with the signing of the July 2012 Food and Drug Administration Safety and Innovation Act (FDASIA). In January 2013, Vertex Pharmaceuticals is awarded the 1st two FDA Breakthrough Therapy Designations for orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF).

Before FDASIA, the FDA had 3 programs to expedite the development and review of drugs for life threatening and serious diseases – Fast Track, Accelerated Approval, and Priority Review. The new FDA Breakthrough Therapy Designation is described in detail in Section 902 of FDASIA. 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.

According to a article :

  1. Pharmaceutical companies may win approval from the FDA for breakthrough therapies “after a single round of studies, rather than three, in an effort to speed them to patients”
  2. Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER), said in a February 11, 2013 presentation at a Bloomberg sponsored “State of Health Care 2013” event in New York, that 3 experimental medicines have been assigned the new Breakthrough Therapy designation – 2 to Kalydeco
  3. Ms. Woodcock goes on to say that this will result in companies having closer communication with the FDA to move drugs for serious diseases to market quicker, “potentially with data from an expanded Phase I trial”.

According to a article, besides Vertex Pharmaceuticals’ breakthrough status for 2 drugs for CF, a 3rd drug has been “anointed with the special status”, and sponsors have submitted 18 for review – mostly for cancer.

The FDA plans on releasing a guidance on the Breakthrough Therapy designation in the near future. Currently, drug companies can submit applications to the FDA following the criteria on the FDA breakthrough Therapy website.

References Video titled, “Urgency” on BreakThrough Therapy

BloombergTV Video titled, “FDA Sees More Breakthrough Drugs”

FDA Fact Sheet: Breakthrough Therapies

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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