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Duchenne Lessons: Advocates For Rare Spinal Disease Have Eyes on FDA

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drug development by providing clinical trial recruitment, patient perspective, and helps develop outcome measures and biomarkers—but this help comes regardless of whether Cure SMA has a relationship or gets funding from a drug’s developer, and only after its advisors and board of directors review the request. There are no “exclusive” deals with anyone, Hobby says.

Hobby adds that there are important benefits to working with industry: The patient’s voice is better heard, and Cure SMA can promote collaborations and joint research efforts between companies, which can speed development.

[Updated to include more of Hobby’s comments on pricing] Hobby says Cure SMA has had a longstanding view on orphan drug pricing, which is that “the ability and freedom to price drugs to match the smaller market size is a key incentive to encourage companies to invest their own resources to develop new therapies. And so we make sure that we are not putting up any barriers that would actually reduce this incentive though pushing for lower prices.” Cure SMA will fight for broader access to drugs, though, as it plans to do with the patient-led meeting with the FDA it wants to schedule.

“It is of minimal impact if we get an approval with a drug label that would cover just 5 percent of our patients, no matter what the price,” he says.

The FDA’s internal struggle, and the pressure it faces from advocates, have never been more clear than with eteplirsen. In April, hundreds of patients and their advocates showed up at a meeting of outside medical experts who advise the agency. More than 50 spoke. All but one pleaded for approval of eteplirsen, the scientific case for which revolves largely around one small, 12-patient study that the FDA has had a hard time drawing conclusions from.

At the hearing, William Dunn, director of the agency’s division of neurology products, said that regardless of how urgently a Duchenne drug is needed, and though “emotions [would] run high,” the FDA would not lower its approval standards.

At the end of the meeting, the advisory panel voted against approval of eteplirsen, which typically—though not always—means the FDA will follow suit. Yet Janet Woodcock, the FDA’s top drug evaluator, showed up during the hearing—an uncommon occurrence. She spoke of regulatory “flexibility” and expressed fear that an unwarranted rejection would have “consequences” on Duchenne patients.

The FDA has since delayed a final decision and asked Sarepta for more data— specifically, information from muscle biopsies taken from 13 more patients on eteplirsen.

With an SMA drug likely to enter FDA review soon, advocates have been watching the Duchenne situation closely. “We’re paying a lot of attention to it,” says Cure SMA CEO Hobby. Cure SMA has “active discussions and dialogue” with the Duchenne community and its patient groups, and some of Cure SMA’s board members are involved with Duchenne foundations.

What Hobby is watching for: Will the FDA approve eteplirsen on an “accelerated” basis, or make Sarepta complete an additional trial? Will the agency fall somewhere in the middle and approve eteplirsen for a narrower group of patients?

“Whatever they do there is going to set a precedent for other orphan therapeutics coming through,” Hobby says. “It’s going to have to be the benchmark for other diseases, like SMA.”

It appears the task will be easier for SMA groups than their Duchenne counterparts. Biogen and Ionis stopped a randomized, placebo-controlled Phase 3 trial of nusinersen in type 1 SMA patients early because they said the drug was clearly improving infants’ motor function—the ability to roll, crawl, or control their head.

“We feel like this is a very clinically meaningful result,” says Wildon Farwell, a senior medical director of clinical development at Biogen. “These are motor milestones that a parent, physician, or patient would appreciate.”

But a number of questions remain. How long do the improvements in motor function last, and what happens if they wear off? Do patients continue to improve as they take the drug, which Farwell says would likely be a chronic therapy? Will any safety problems crop up over time? And how much would it cost?

“We still have a lot to learn about the disease,” Farwell says. “And we’ll only understand as we continue to follow these patients over time.”

If Cure SMA is going to fight for a broader label on nusinersen, ongoing studies will be crucial. A Phase 3 trial of nusinersen in childhood-onset SMA, which includes the less common type 2 and 3, should wrap up in the first half of 2017. That study is particularly important for folks like the Tomko sisters, who have type 2. If nusinersen doesn’t produce positive results, they might not be eligible for it.

Both Jessica and Heather Tomko admit that they haven’t paid close attention to nusinersen’s development, since it’s unclear whether it’ll ever impact them personally. But Heather has started to work with Cure SMA on some fundraising, even if it’s hard given the impact her disease has had on her. She says she knows more about SMA and how it affects people than the doctors she sees. She knows what works, what her body needs, when she’s getting sick, and what to do about it.

Perhaps, if nusinersen shows even a slight benefit for type 2 SMA patients, that’s a story she’ll tell at an FDA hearing some day.

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