Sangamo's ST-920 BLA Pathway
Sangamo Therapeutics, Inc. announced important derisking events in the pathway to a planned BLA submission for isaralgagene civaparvovec, or ST 920, its wholly owned gene therapy product candidate for the treatment of Fabry disease.
All dosed patients in the Phase I/II STAAR study evaluating isaralgagene civaparvovec have now completed at least 52-weeks of follow-up, a key milestone required by the FDA for an Accelerated Approval regulatory pathway for ST 920. Preliminary analysis of clinical data collected as of this 52-week milestone date across all 32 dosed patients indicates that the mean eGFR slope continued to remain positive, following the last clinical update at the WORLDSymposium in February 2025, with a data cutoff date of September 12, 2024. The product candidate continues to be well tolerated. A pivotal data readout is expected by the end of the second quarter of 2025.
Furthermore, in April 2025, Sangamo held a productive Type B meeting with the FDA, providing Sangamo with a clear CMC pathway to a planned BLA submission in the first quarter of 2026, including clarity on plans for process validation, path to commercial specifications and the commercial launch manufacturing site. This BLA submission timeline would facilitate a potential approval and commercial launch as early as the second half of 2026.
“Following last year’s alignment with the FDA on an Accelerated Approval regulatory pathway for ST-920, we are excited to have now gathered the one-year mean eGFR slope data that will serve as the primary efficacy endpoint for our planned BLA submission,” said Nathalie Dubois-Stringfellow, Ph. D., Chief Development Officer at Sangamo. “Coupled with our recent productive FDA Type B meeting, we have a clear regulatory pathway to a potential approval decision for ST-920 and we continue to advance BLA preparation activities.”
Discussions with the European Medicines Agency (EMA) on the proposed pathway to potential approval for isaralgagene civaparvovec in Europe are ongoing. In addition, Sangamo continues to engage in business development negotiations for a potential Fabry commercialization agreement.
About the STAAR Study The Phase 1/II STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate the safety and tolerability of isaralgagene civaparvovec, or ST 920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. The STAAR study enrolled male and female patients who are on ERT, are ERT pseudo-naïve (defined as having been off ERT for six or more months), or who are ERT-naïve. The FDA has granted Orphan Drug, Fast Track and RMAT designations to isaralgagene civaparvovec, which has also received Orphan Medicinal Product designation and PRIME eligibility from the EMA and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.