SuperTalk Mississippi

Sen. Wicker helps with fight for first drug to treat Duchenne Muscular Dystrophy

WASHINGTON, DC – Following efforts from U.S. Senators Roger Wicker, R-Miss., and Amy Klobuchar, D-Minn., the Food and Drug Administration (FDA) has announced that it has accelerated approval for the first drug to treat Duchenne Muscular Dystrophy (DMD).


In April, Wicker and Klobuchar penned a bipartisan letter urging the FDA to use all available resources and authorities to accelerate the process of getting safe and effective treatments to patients diagnosed with DMD.


In a statement, the Senators also highlighted the Food and Drug Administration Safety and Innovation Act (FDASIA), legislation that provides the agency with improved flexibility to grant approval to rare disease treatments that have proven to be beneficial and allows the FDA to impose post-approval studies to confirm the clinical benefit.


The Patient Focused Drug Development initiative, also included in the law, asks that the agency consider the views and experiences of patients as part of the drug review process.


“In the 15 years since I introduced the MD-CARE Act, this ranks as one of the most important milestones in our fight to cure Duchenne,”Sen. Wicker said. “I am thrilled that the FDA has acted within its authority to allow young men and their families to have access to this drug. We should take every opportunity to help improve the quality of life for those living with the disease.”
“Today’s announcement will make a tremendous difference for people living with Duchenne muscular dystrophy. I am proud that through our bipartisan efforts, the Food and Drug Administration was able to use all available tools at their disposal to approve this groundbreaking drug,” said Sen. Klobuchar, a Senate co-chair of the Rare Disease Congressional Caucus. “We are now one step closer to improving the quality of life for those living with this disease.”
In 2014, Wicker and Klobuchar’s legislation to help improve the lives of patients with muscular dystrophy was signed into law by the President. The Muscular Dystrophy Community, Assistance, Research & Education (MD CARE) Act supports medical research and policies to improve treatments and quality of life for muscular dystrophy patients.

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