Sangamo Therapeutics Fast-Tracks Fabry Gene Therapy with FDA Accelerated Approval Pathway

Accelerated Approval Pathway:
Sangamo Therapeutics has aligned with the FDA on an accelerated approval pathway for its Fabry disease gene therapy candidate, isaralgagene civaparvovec (ST-920), potentially shortening the time-to-market by three years.

Clinical Data:
The FDA agreed to use estimated glomerular filtration rate (eGFR) slope data at 52 weeks from the Phase 1/2 STAAR study as the primary basis for accelerated approval.

Submission Timeline:
The complete dataset to support accelerated approval will be available in the first half of 2025, with a potential Biologics License Application (BLA) submission expected in the second half of 2025.

STAAR Study:
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study evaluating the safety and tolerability of isaralgagene civaparvovec in patients with Fabry disease.

Regulatory Designations:
Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as Orphan Medicinal Product designation and PRIME eligibility from the EMA.

Business Development:
Sangamo is seeking a development partner for the gene therapy and has initiated BLA readiness activities while continuing business development discussions.