DMD: Drisapersen Receives ‘Breakthrough Therapy’ Designation

The FDA will speed up the review process for a promising exon-skipping drug designed to treat Duchenne MD caused by specific dystrophin mutations

The FDA’s “breakthrough therapy” designation provides more guidance from the agency to the drug company regarding the regulatory process, as well as faster review of promising therapeutics.
Article Highlights:
  • The FDA has given "breakthrough therapy" designation to GlaxoSmithKline's experimental Duchenne MD drug drisapersen, which is designed to cause skipping of exon 51 of the dystrophin gene and lead to production of dystrophin, the muscle protein missing in this disease.
  • Breakthrough therapy designation provides increased FDA guidance and speeds the review process for investigational drugs that meet certain criteria.
  • Clinical trials of drisapersen are ongoing.
by Margaret Wahl on July 3, 2013 - 9:51am

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

Drisapersen is one of two experimental DMD drugs currently in clinical trials that works by a mechanism known as exon skipping. It is designed to allow production of the needed dystrophin protein in the muscles of boys with DMD caused by specific mutations and has shown promising results in a phase 2b clinical trial.

(The other DMD exon-skipping drug currently in clinical trials is eteplirsen, from Sarepta Therapeutics. Both drugs target a region of the dystrophin gene known as exon 51.)

About the FDA designation

FDA breakthrough therapy designation is a mechanism designed to expedite the development and review of drugs intended to treat a serious condition and for which preliminary evidence indicates that the drug may demonstrate substantial improvement over available therapy. It's one of several mechanisms the FDA has developed to speed up the review process for certain types of new medications.

A company whose drug is designated a breakthrough therapy gets advice and meetings from the FDA early in the drug development process, involvement of senior FDA staff members and other experts, and the ability to submit parts of the new drug application as they become finalized.

Drisapersen clinical trials are ongoing (see A Snapshot View: Drisapersen and Eteplirsen to learn more).

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