PTC Therapeutics expects FDA BLA filing for ST-920 Fabry gene therapy in Q4 2026 - PTCT News | RalliesPTC Therapeutics expects FDA BLA filing for ST-920 Fabry gene therapy in Q4 2026
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PTCT• Deal terms
- Deal terms include USD 111 million upfront, up to USD 100 million in regulatory milestones, with closing expected in late Q3 or early Q4 2026.
FDA filing and launch timing
- Rolling FDA filing targets accelerated approval, with submission expected to be completed in Q4 2026 and a potential 2027 launch.
PTC Therapeutics to acquire ST-920 gene therapy
- PTC Therapeutics won a bankruptcy auction to acquire Sangamo’s BLA-stage ST-920 gene therapy for Fabry disease.
- Phase 1/2 STAAR results were already presented, showing sustained clinical benefit and a tolerable safety profile that supported stopping chronic enzyme therapy.
