Stoke Therapeutics announced that its request for an expedited filing of the severe epilepsy drug, zorevunersen, was not approved by the Food and Drug Administration (FDA). The decision came after a meeting held in December 2023, where the FDA did not outright reject Stoke’s proposal. Instead, the agency requested additional information before further discussions can take place.
Ian Smith, CEO of Stoke Therapeutics, explained that while the FDA has not closed the door on the possibility of submitting zorevunersen later this year, the company must first complete an ongoing Phase 3 study, which is expected to conclude in mid-2027. This study focuses on zorevunersen as a treatment for Dravet syndrome, a severe form of epilepsy that can lead to significant developmental challenges in children.
According to Smith, the FDA’s request for more information indicates that regulators are open to dialogue regarding the regulatory path for zorevunersen. The company plans to provide the requested data and expects to determine its course of action by mid-2024.
The FDA’s decision reflects the agency’s rigorous standards for drug approval, particularly for treatments addressing complex conditions such as Dravet syndrome. Stoke Therapeutics remains committed to advancing its research and is hopeful that continued collaboration with the FDA will facilitate a timely resolution.
The outcome of this regulatory discussion is crucial for patients suffering from Dravet syndrome and their families. Current treatment options remain limited, and the potential approval of zorevunersen could provide a much-needed alternative for managing this challenging condition.
As Stoke Therapeutics prepares for its next steps, the company emphasizes its focus on delivering innovative therapies that can significantly improve the quality of life for patients with severe epilepsy. The coming months will be critical for both the company and the families awaiting advancements in epilepsy care.
