Second chance for DMD drug gives hope

The FDAs decision to give an experimental drug for Duchenne muscular dystrophy another chance is rekindling hope in parents in the Bay State and beyond that they might be able to save their children from the worst effects of the fatal disease.

The Food and Drug Administration has cleared the way for Cambridge-based Sarepta Therapeutics to apply for approval of eteplirsen, a drug that treats DMD. Its a move Sarepta has been awaiting and DMD families have been demanding.

This is certainly a step in the right direction, said Tracy Seckler, whose son Charley suffers from the muscle-wasting disease. Its hard to express extreme joy at the FDA doing its job properly and doing what should have been done already, but under the circumstances its encouraging.

The FDA previously had stalled approval of eteplirsen over concerns about its safety and efficacy. The drug has been tested in only 12 boys, but it appears to be effective for them. Boys with DMD lose mobility over time and typically dont live past their 20s. There are about 18,000 to 20,000 DMD patients in the United States.

The FDA is fully committed to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible, and is actively engaged with all drug companies developing new drugs for Duchenne muscular dystrophy, FDA spokeswoman Sandy Walsh said yesterday.

Sarepta is planning four clinical studies on dozens of patients at different stages of DMD, including young boys who are less affected, and teens who have lost their ability to walk.

Pembroke mother Jenn McNary hopes her son, Austin Leclaire, 15, now will be part of a trial. Her younger son, Max Leclaire, 12, also has DMD and is part of the original eteplirsen study, which the family believes has helped him maintain mobility.

It was a really big win, McNary said of the FDAs decision, but the kids are still not receiving the treatment. Its still words on paper. This has taken so long that (Austin has) lost function he cant get back, she said.

Austin uses a wheelchair and is losing upper body strength.

Sarepta officially will file a new drug application with the FDA later this year. If the FDA gives the application a priority review and ultimately approves it, eteplirsen could be commercially available in the second half of 2015, Sarepta spokesman Jim Baker said.

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Second chance for DMD drug gives hope