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FDA denies approval of muscular dystrophy drug; company plans to appeal

New Haven Register

10-26-17

Oct. 26--NEW HAVEN -- The U.S. Food and Drug Administration has ruled against approving a drug for muscular dystrophy that a student at Southern Connecticut State University relies on to live independently and to walk long distances without tiring, according to the drug's manufacturer, PTC Therapeutics.

PTC plans to appeal the ruling, which calls for PTC to conduct an additional clinical trial in order for the drug, ataluren, to be considered for approval, according to a press release issued Wednesday.

Josh Wagner, 24, has Becker muscular dystrophy, a less severe form of MD than Duchenne, for which ataluren specifically has been targeted.

"We are extremely disappointed for the Duchenne community and strongly disagree with the agency's conclusions," said Stuart W. Peltz, chief executive officer of PTC Therapeutics, in a statement. "We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week."

According to Jane Baj, a spokeswoman for PTC, which is based in South Plainfield, New Jersey, the appeal would go through a different FDA office than the one that rejected approval of ataluren, the Office of Drug Evaluation I.

"We are filing next week and we're just going to work through that process," Baj said.

According to PTC, the FDA's "complete response" 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."

Ataluren is marketed under the trade name Translarna in the European Union, Iceland, Liechtenstein, Norway, Israel and South Korea, according to the company's website. "We have been providing it for at least 10 years ... for 150 boys in the United States and we will continue to do so," Baj said. Ataluren has only been available in the United State to those enrolled in clinical studies. Two worldwide trials have been conducted involving 400 patients, Baj said.

Wagner, 24, of Meriden, at one time was so weak he needed a wheelchair or motorized scooter to go more than a couple of blocks or his leg muscles would become tight and stiff. Since taking ataluren, he has been able to complete a 5K run and has begun lifting weights. An accounting major, he will graduate from Southern in May.

Wagner could not immediately be reached for comment.

Sandy Walsh, a spokeswoman for the FDA, said in an email, "the FDA recognizes the unmet medical need of patients with Duchenne muscular dystrophy, a devastating disease for patients and their families. We remain committed to addressing the urgent need for new treatment options. We have taken a number of steps in recent months to advance and support the development of orphan drugs, and will continue to look for opportunities to specifically support the advancement of DMD treatments."

Walsh said the FDA has approved two treatments for Duchenne, "but we understand that much more is needed and recognize the importance of bringing new treatment options to this community."

One of those treatments is eteplirsen, marketed as Exondys 51, which Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, approved over the objections of her staff.

An FDA advisory committee, after hearing testimony from Wagner and others and taking in hundreds of comments, voted 10-1 against approving ataluren.

Ataluren works by helping restore the protein dystrophin, which those with Duchenne muscular dystrophy lack because of a "nonsense mutation" in their genes, according to PTC. Those with Becker MD do not completely lack dystrophin, and their skeletal muscles are not affected as severely, but their heart muscles may be weakened similarly to those with Duchenne, according to the Muscular Dystrophy Association's website.

Contact Ed Stannard at edward.stannard@hearstmediact.com or 203-680-9382.

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