Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies

Leading Duchenne-Specific Nonprofit Urges Congress to Enact as Part of User Fee Bill

HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) the leading advocacy organization fighting to end Duchenne muscular dystrophy has endorsed legislation that would shorten the amount of time the Food and Drug Administration (FDA) takes to review candidate therapies for life-threatening conditions like Duchenne that lack other treatment options.

(Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO)

The bill, known as the Faster Access to Specialized Treatments or FAST Act, is bipartisan legislation sponsored by Rep. Cliff Stearns of Florida and Rep. Edolphus Towns of New York.

"For 20 years, FDA has had tools at its disposal to accelerate the agency's review of therapies targeted to treat life-threatening conditions that lack any other therapeutic options. Unfortunately, these tools have not yielded optimal advances despite the significant need that exists for patients and families living with Duchenne and other conditions," Pat Furlong, Founding President and CEO of PPMD, said.

"The FAST Act is commonsense legislation that would leverage the Fast Track and Accelerated Review options at FDA's disposal. It would allow sponsors of candidate drugs to petition FDA to designate their product as being eligible for Fast Track review. It would also allow for approval of therapies on the condition of surrogate endpoints or intermediate markers used to gauge the trajectory of a disease," Furlong added.

Surrogate endpoints are a particularly important issue to the Duchenne community given the course of the disease and the need to more quickly determine whether or not a therapy is having its intended effect.

The legislation also seeks to more appropriately balance the benefits of a therapy with the potential risks. It would revoke accelerated approval if a sponsor fails to adequately study and monitor the therapy in patients or if further studies fail to verify clinical benefit.

Original post:
Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies