BEDFORD, MA - Stoke Therapeutics, Inc. (NASDAQ:STOK) has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for its drug candidate zorevunersen, aimed at treating Dravet syndrome, a rare form of epilepsy. This designation is pivotal for the biotechnology firm as it propels the drug closer to potential approval and market availability.
Zorevunersen, which is on course to be the first disease-modifying treatment for Dravet syndrome, targets a confirmed mutation in the SCN1A gene that does not involve gain-of-function. Dravet syndrome patients suffer from severe seizures that are often resistant to current medications.
The FDA's decision comes on the heels of positive outcomes from Phase 1/2a and open-label extension (OLE) studies, which showed that zorevunersen significantly reduced seizure frequency and improved cognitive and behavioral measures in patients. These benefits were noted in addition to the effects of existing anti-seizure medications. The drug has been generally well-tolerated, with over 600 doses administered, some patients receiving treatment for over three years.
Stoke Therapeutics experienced a 2.1% increase in its shares in premarket trading following the announcement.
The company is in ongoing discussions with the FDA and other global regulatory bodies to advance zorevunersen into a global, randomized, controlled Phase 3 registrational study. An update on the Phase 3 plans is expected by the end of the year.
Shamim Ruff, Chief Regulatory Affairs Officer at Stoke Therapeutics, expressed gratitude towards the FDA for the designation and highlighted the clinical data that suggest zorevunersen could substantially improve upon current Dravet syndrome treatments by addressing the underlying cause of the disease.
The Dravet Syndrome Foundation, represented by Executive Director Mary Anne Meskis, also welcomed the designation, emphasizing the urgency for innovative treatments that go beyond symptom management to address the root cause of Dravet syndrome.
The Breakthrough Therapy designation is designed to expedite the development and review process for drugs intended to treat serious conditions when preliminary clinical evidence indicates the potential for significant improvement over existing therapies. It provides benefits such as Fast Track designation features, intensive guidance on efficient drug development, and involvement of senior FDA managers.
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