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Duchenne advocates’ wait for an FDA decision nears the six-month mark

One is hiking up the highest mountain in Malaysia to raise awareness for the disease. One has spent the last couple months vetting drug trials that are now enrolling across the U.S. One is just trying to remain optimistic in the face of a complete lack of news from the company as well as the federal agency that holds the near-term fate of thousands in its hands.

It’s been nearly six months since the Food and Drug Administration’s original Feb. 26 deadline to make a decision whether to approve the experimental drug for Duchenne muscular dystrophy by Cambridge-based Sarepta Therapeutics (Nasdaq: SRPT). That date has been pushed back repeatedly — first due to a snowstorm, then due to the FDA’s “continuing review and internal discussions” of the scientific data surrounding the drug, called eteplirsen.

Currently, no one appears to know when a decision will be forthcoming. Rumors that the company may be presenting at the Morgan Stanley Global Healthcare Conference 2016 next month — which would suggest the company is expecting its “quiet period” pending the FDA’s decision could end soon — are as yet unconfirmed (the company isn’t commenting). Likewise, speculation that the holdup at the FDA is due to disagreement between higher-ups at the agency and the specific division that conducted the review of the drug are also just that — speculation.

Asked about the ongoing delay and the effect it’s having on patients and their families, the FDA’s head of drug approvals, Janet Woodcock, said she’s bound by law not to comment. But patient advocates themselves have told me in recent days that the wait is taking a toll. Beyond the hundreds of boys with Duchenne who have died, been forced to use wheelchairs or lost their ability to feed themselves in the past few months, Christine McSherry, head of the Massachusetts-based advocacy group, the Jett Foundation, said that many patients are refusing to enroll in other drug studies until a decision on eteplirsen has been rendered.

“There are lots of kids out there who could start another trial,” said McSherry, a Pembroke resident. But many aren’t, she said, for fear that they’d have drop out if the drug is approved, and then may need to wait additional weeks for any other drug to flush out of their system before being eligible to take eteplirsen.

“What would happen if they should enroll their child in another trial?”

McSherry — whose 19-year-old son, Jett, has the disease — said local companies like Catabasis Pharmaceuticals (Nasdaq: CATB) are currently enrolling in Duchenne trials, as are San Francisco–based FibroGen and Beverly Hills, California-based Capricor Therapeutics. “We’ve been doing due diligence on these trials to make sure people know about them,” she said.

Cath Jayasuriya, founder and executive director of Newport Beach, California-based Coalition Duchenne and the mother of a 23-year-old with Duchenne, said in an email, “The delay in making a decision ignores the tremendous cost to boys and young men with Duchenne and their families.”

Today, Jayasuriya is in Borneo for her foundation’s 6th annual Expedition Mt. Kinabalu, a way of raising awareness for Duchenne. “Here we are launching a new initiative aimed at underserved boys in rural areas and helping where ever we can,” she said.

But Debra Miller, the founder of another disease foundation that also happens to be in Newport Beach called CureDuchenne, says she’s choosing to take the glass-half-ful approach.

“Every single day that we don’t have a denial is a good day,” she said. She believes that “there’s a lot going on behind the scenes” at the FDA, and says, “I think they really want to approve the drug.”

Still, she said the lack of a specific timeline is frustrating. “It’s wearing on the community for sure,” she said.

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