PYC - General Discussion, page-679

US biotech, Stoke Therapeutics, has been granted Breakthrough Therapy Designation (BTD) by the FDA for zorvunersen for the treatment of Dravet syndrome. Zorevunersen, which is being developed as potentially the first disease-modifying therapy in Dravet syndrome, is Stoke’s lead compound and currently its only drug in clinical trials.

Stoke Therapeutics is focused on developing antisense oligonucleotide therapies for monogenic diseases of autosomal dominant haploinsufficiencies.

The designation was granted on the basis of Phase 1/2a and open-label extension data which demonstrated substantial and sustained reductions in seizure frequency and continuous improvements in multiple measures of cognition and behaviour.  An update on Stoke’s Phase 3 registrational plans is expected later this month.

BTD grants zorevunersen access to all FDA Fast Track designation features, intensive guidance on an efficient drug development program and an organizational commitment involving senior FDA managers. Recently published analysis suggests that the BTD program lowers late-stage clinical development time by 30 %.

Drug sponsors are advised that BTD requests should be submitted no later than the end-of-Phase-2 meetings. The FDA provides a response within 60 days.

STOK shares rose over 12% on the news.

https://finance.yahoo.com/news/stoke-therapeutics-receives-fda-breakthrough-120000759.html

https://www.stoketherapeutics.com/scientific-platform/

https://www.fda.gov/patients/fast-t...approval-priority-review/breakthrough-therapy

https://www.precisionformedicine.com/blog/what-is-an-fda-breakthrough-therapy-designation

https://www.healthaffairs.org/doi/abs/10.1377/hlthaff.2023.00837

https://www.cas.org/resources/cas-insights/breakthrough-therapy-designation-real-world-impact