Drugs

Stoke, FDA Align on Planned NDA for Dravet Syndrome Drug

Stoke Therapeutics says it has reached agreement with FDA on key elements of a planned NDA submission for zorevunersen, an investigational antisense oligonucleotide for Dravet syndrome. The company says it is targeting a submission in the the second half of 2027.

A recent FDA and company meeting covered five years of safety and efficacy data from its Phase 1/2a and open-label extension studies, the timing and contents of the planned application, and analysis of a key secondary endpoint in the Phase 3 EMPEROR trial, according to Stoke

Following FDA feedback, Stoke plans to evaluate changes in cognition and behavior using a multicomponent assessment of four prespecified Vineland-3 subdomains: expressive communication, receptive communication, interpersonal relationships and personal skills. The data will be analyzed using a multivariate mixed model for repeated measures, it says.

Stoke CEO Ian Smith says FDA is willing to accept the multicomponent assessment in the NDA, which the company believes will better capture the drug's potential effects on the range of neurodevelopmental problems associated with Dravet syndrome.

The Phase 3 EMPEROR trial is comparing intrathecal zorevunersen with a sham procedure over 52 weeks. Its primary endpoint is change in major motor seizure frequency at Week 28, with durability of seizure reduction and changes in adaptive functioning among key secondary endpoints, Stoke says.

Stoke expects the Phase 3 readout in the third quarter of 2027 and plans to complete the NDA submission shortly afterward in the second half of the year, potentially supporting U.S. approval and launch in early 2028.

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Sharfstein et al. Urge Stronger FDA Oversight of Accelerated Approvals

FDA Actions Signal Scrutiny of Telehealth Prescribing Models: Attorneys

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TikTok Misinformation on Acetaminophen, Autism Surged After FDA Notice