Sangamo Advances FDA Rolling Submission for Fabry Disease Gene Therapy — Sangamo Therapeutics, Inc.

Event summary

Sangamo Therapeutics has advanced the rolling submission of a Biologics License Application (BLA) to the FDA for ST-920, its gene therapy for Fabry disease.
The Phase 1/2 STAAR study demonstrated positive mean annualized eGFR slope at 52-weeks across all dosed patients, supporting an accelerated approval pathway.
The antibody assay companion diagnostic has been submitted to and accepted by the FDA’s Center for Devices and Radiological Health (CDRH) seeking Premarket Approval (PMA).
ST-920 has received Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as similar designations from European and U.K. regulators.

The big picture

Sangamo's advancement in the rolling submission of a BLA for ST-920 marks a significant step towards potentially shifting the treatment paradigm for Fabry disease, a rare lysosomal storage disorder. The positive clinical data from the STAAR study supports the potential of isaralgagene civaparvovec as a one-time, durable gene therapy option. This development comes at a time when the biotech industry is increasingly focused on gene therapies for rare diseases, with regulators showing willingness to expedite approvals through accelerated pathways.

What we're watching

Regulatory Pathway: Whether the FDA will accept the 52-week eGFR slope data as sufficient for accelerated approval.
Clinical Efficacy: The durability of the therapeutic effects observed in the Phase 1/2 STAAR study and whether final clinical trial data will validate the safety and efficacy of isaralgagene civaparvovec.
Commercialization Strategy: Sangamo’s ability to secure a commercialization partner for ST-920, given its lack of capital resources to obtain regulatory approval and commercialize the product candidate on its own.