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FDA Says PTC Therapeutics’ New Drug Application for DMD Treatment Cannot Be Approved in its Current Form

Press Release: PTC Therapeutics Receives Complete Response Letter for Ataluren’s NDA

Summary: PTC Therapeutics reported on Oct. 25, 2017, that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) indicating that the Office of Drug Evaluation of the FDA is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form. Specifically, the letter indicated that evidence of effectiveness from an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness.

Stuart W. Peltz, chief executive officer of PTC Therapeutics indicated in the press release that they believe the FDA’s decision doesn’t consider the benefit-risk of Translarna and that the company plans to file a formal dispute resolution next week.

Statement from MDA President and Chief Executive Officer Lynn O’Connor Vos:

“Although today’s decision does not result in the addition of another available treatment option for Duchenne muscular dystrophy, it reinforces the commitment all of us at MDA have to find solutions for our families.

“Since 2015, the MDA community has seen six drug approvals for neuromuscular diseases in our program, including two approvals for drugs to treat DMD. While new treatments and cures can’t come fast enough for our community, we are hopeful as more experimental therapies than ever before are reaching late-stage development and moving through the regulatory review process. We remain firmly committed to working together with the DMD community at such a promising time.

“MDA is the world leader in applying innovation and cutting-edge technology to the development of therapies for neuromuscular diseases, and we will continue to fund the best scientific research, encourage participation in clinical trials, and advocate for policies that facilitate drug development, as we work to make safe and effective treatment options available for individuals living with neuromuscular disease.”

Background: Translarna is under development by PTC Therapeutics to treat DMD caused by a type of genetic mutation known as a “nonsense mutation” or “premature stop codon.” This type of mutation causes the muscle cell to stop reading the genetic code used to manufacture the muscle protein dystrophin before it reaches the end of the genetic instructions, thus preventing production of the protein. A “readthrough” drug, Translarna is designed to obscure the incorrect stop signal and thereby restore production of dystrophin. The drug, which is taken by mouth, may help approximately 13 percent of people with DMD.

MDA has funded groundbreaking DMD research for more than 65 years, including laboratory and clinical testing of Translarna and other stop codon read through therapies. MDA is proud of our decades-long approach to providing coordinated multidisciplinary care to individuals living with DMD through our nationwide network of more than 150 MDA Care Centers. We have facilitated direct patient care to tens of thousands of individuals living with DMD over more than half a century.