WASHINGTON, April 19, 2016 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), commended the House Appropriations Committee for including language within the Fiscal Year 2017 FDA spending bill that recognizes the value and importance of intermediate clinical endpoints in regulatory reviews.
The language, requested by Reps. Peter King (R-NY), Doris Matsui (D-CA) and nearly 50 additional House members, was championed by PPMD during the group's advocacy conference earlier this year. It is included in the FY 2017 Agriculture, FDA and Related Agencies appropriations bill, expected to be approved by the full House Appropriations Committee later today.
"PPMD is very grateful to Chairman Aderholt and Ranking Member Farr for including this important language in their committee report," said PPMD Founding President & CEO Pat Furlong. "This language simply recognizes that Congress has provided FDA with the legal authorities and tools needed to use intermediate clinical endpoints in evaluating candidate therapies for Duchenne and other rare diseases."
Following is the report provision:
Duchenne Muscular Dystrophy—The Committee is encouraged that the FDA has the tools, authorities, and latitude necessary to review and approve safe and effective treatments for rare diseases, such as Duchenne Muscular Dystrophy, as efficiently as possible. In particular, the Committee is aware that the use of intermediate clinical endpoints (ICE) may be an appropriate approach as it has been in similar deadly diseases with dire unmet needs, such as HIV and cancer.
Intermediate clinical or surrogate endpoints were critical to the successful efforts two decades ago to develop treatments for HIV/AIDS where no alternatives existed, and have also been used in other disease areas. Next week, the FDA will convene an advisory committee meeting focused on a candidate therapy to treat a form of Duchenne.
"Congress has recognized for years that for patients with fatal diseases that lack any other treatment alternatives, time is of the essence. This provision builds upon this longstanding recognition, one we hope FDA reviewers will embrace as warranted going forward," Furlong said.
The language follows a bipartisan Senate letter, led by Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) and signed by 22 other Senators encouraging agency reviewers with a similar message.
About Parent Project Muscular Dystrophy Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.