PPMD Applauds FDA for Landmark Approval of First-Ever Disease-Modifying Drug to Treat Duchenne Muscular Dystrophy

HACKENSACK, N.J., Sept. 19, 2016 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), heralded today's decision by the Food and Drug Administration (FDA) to approve the first-ever U.S. application for a drug to treat Duchenne.

The agency's decision to approve Exondys 51 (eteplirsen) – a therapy specifically indicated for people living with Duchenne who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping – under the Accelerated Approval pathway, caps more than a year of agency review.

"PPMD is thrilled that FDA has granted an Accelerated Approval to Exondys 51, a therapy with the potential to treat 13% of people living with Duchenne, marking the first-ever U.S. approval of a drug to treat this disease," said PPMD Founding President and CEO Pat Furlong.

"We applaud Sarepta and the many clinicians and scientists who continue to work tirelessly on this therapy. And we are eternally grateful to all of the young men, and their families, who participated in this trial, and the organizations and advocates who worked collaboratively to make this historic day possible. Their sacrifices have forever changed the Duchenne landscape.

"This milestone comes nearly 15 years after the enactment of the landmark Muscular Dystrophy CARE (MD-CARE) Act that PPMD and the entire community fought so hard to achieve. The MD-CARE Act stimulated basic research that led us to this success.

"We stand ready to assist the company in fulfilling its post-approval requirements. We want to commend FDA leadership for recognizing that Exondys 51 has a favorable benefit-risk profile and that it met its Accelerated Approval requirements. This approval demonstrates an agency commitment to the needs of patients, particularly those with serious unmet medical needs," Furlong added.

Under the approval, Sarepta Therapeutics will be required to conduct a clinical trial to confirm the drug's clinical benefit. The required study will be designed to assess whether Exondys 51 improves motor function of Duchenne patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. The primary endpoint will be the North Star Ambulatory Assessment.

Going forward, PPMD remains focused on ensuring all patients who are candidates for eteplirsen obtain affordable access to the drug while it continues to support the development of additional tools and resources to drive forward approvals of additional therapies and treatments.

One of the tools that is vital in driving forward new advances in therapies and treatments is the PPMD patient report registry, DuchenneConnect. DuchenneConnect was established in 2007 to serve as a resource for the community by bringing together those living with the disease and connecting them to research, clinical care, and clinical trials. DuchenneConnect is also a resource for researchers and industry, allowing access to aggregated, de-identified information provided by patients and families. This information has proven vital to advances in care and treatment.

With the help of Sarepta, PPMD and DuchenneConnect launched Decode Duchenne in 2013 – a program that offers free genetic testing to people with Duchenne or Becker muscular dystrophy who otherwise could not afford genetic testing. Sarepta continued its involvement during the expansion of the Decode Duchenne program, with the help of other industry partners in 2015-2016. This expansion allowed more testing and counseling to those previously unable to access testing due to financial constraints. PPMD looks forward to a continued partnership with Sarepta and other industry leaders for the next phase of Decode Duchenne, so all people with Duchenne and Becker muscular dystrophy have access to genetic testing and counseling.

To support additional product development and approval, PPMD is building upon its sector-leading patient engagement efforts that have included patient preference studies and a community-generated draft guidance document to conduct additional preference studies and to develop other tools that can advance the process. PPMD has also served as the leading advocacy organization driving national Duchenne newborn screening efforts and policy efforts around access and reimbursement in anticipation of this remarkable day – the first approved therapy for Duchenne.

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 people 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 people with Duchenne live longer, stronger lives. We will not rest until every person 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. Follow PPMD on Facebook, Twitter, and YouTube.

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SOURCE Parent Project Muscular Dystrophy

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