Parent Project Muscular Dystrophy

PPMD is headquartered in Middletown, Ohio.

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

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.


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.

"With nearly 20 potential therapies in clinical testing, it is a very exciting time for the Duchenne community, but a time in which we must make sure the FDA is equipped with the tools and laws it needs to evaluate these therapies as quickly as possible to save our sons," Furlong said.

"I commend Congressmen Stearns and Towns for introducing this legislation, and I urge other members to cosponsor it today so it can become law this year," she added.

The FAST Act is consistent with the FDA policy position the PPMD Board of Directors adopted earlier this year, that seeks to address critical issues impacting the Duchenne and rare disease communities. PPMD is evaluating other FDA-related legislative proposals and engaging with Members of Congress on issues unaddressed to date, including the importance of making sure the voices of rare disease patients are heard when critical decisions, such as those about clinical trial design and appropriate levels of benefit and risk, are being made.

About DuchenneDuchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function.

About Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. Our mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information, visit www.ParentProjectMD.org .

SOURCE Parent Project Muscular Dystrophy