FDA’s Woodcock on developing neuromuscular drugs: ‘We have to change our approach’

As more innovative drugs emerge for various forms of neuromuscular disease, the Food and Drug Administration is under increasing pressure to approve the treatments. Patients are understandably clamoring for cures, and the increasing demands from families have prompted lawmakers to scrutinize agency initiatives. Signs of the growing pressure were evident two years ago in the ruckus over the FDA’s approval of a medicine for Duchenne muscular dystrophy. Meanwhile, these treatments are being launched at increasingly high price points. On the eve of a Muscular Dystrophy Association conference, we spoke briefly with Dr. Janet Woodcock, who heads the Center for New Drug Evaluation and Research at the FDA, about agency moves to hasten approvals. This is an edited version of the conversation…

Pharmalot: In your view, how far have we come recently?

Woodcock:  I think there’s really been progress in neuromuscular disease. This was a backwater for a very long time and patients didn’t have any treatment or hope on the horizon. But we’ve approved a number of drugs from a number of novel platforms recently. For spinal muscular atrophy, we approved a drug that was effective and now it’s available [this was Spinraza, a Biogen (BIIB) drug that was endorsed by the agency in late 2016]. We also approved Onpattro in August… that is a first-in-class drug that fights fatal neuropathy. And we just approved a drug for primary progressive multiple sclerosis. The point is the pipeline is pretty full in the neurodegenerative space.

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