U.S. Sen. Roger Wicker (R-MS) led a congressional call on Friday for the FDA to use a new approval pathway to advance treatments for Duchenne Muscular Dystrophy (DMD).
Under the Food and Drug Administration Safety Innovation Act (FDASIA), the FDA was given flexibility to approve treatments for rare diseases and to impose post-approval studies that confirm clinical benefits.
In a letter to Dr. Janet Woodcock, the director of the FDA’s Center for Drug Evaluation and Research, Wicker and U.S. Sen. Amy Klobuchar (D-MN) called on the FDA to use all available resources to expedite approval of treatments for DMD.
“For those living with Duchenne and their families, every minute matters,” Wicker said. “Over the past two decades, researchers and medical professionals have made significant strides in the fight against this fatal disease. With modern advancements in science, we have every reason to continue the march toward even better treatments and ultimately a cure. Congress has given FDA new tools to accelerate these life-changing goals. I am hopeful that the agency will leave no stone unturned.”
Under the Patient Focused Drug Development initiative, which was included in the FDASIA, the FDA is allowed to consider the views and experiences of patients during its drug review process.
“For families living with Duchenne Muscular Dystrophy, any advancement of treatments can make a real difference in quality of life,” Klobuchar said. “While we’ve made progress in recent years, much more needs to be done to help patients with this rare but deadly disease. I’m hopeful the Food and Drug Administration heeds our bipartisan call to use all available tools at their disposal to help those with Duchenne by moving safe and effective treatments forward.”
The bipartisan letter to Woodcock, which was signed by 24 senators, including U.S. Sens. Shelley Moore Capito (R-WV), Bill Cassidy (R-LA) and Susan Collins (R-ME), concluded that, “Treatments that are safe and reasonably likely to produce clinical benefit for DMD patients could meaningfully alter their lives.”