Sangamo recently had a meeting with the FDA about their gene therapy product, ST-920, which is being developed to treat Fabry disease. The FDA has agreed that data from the Phase 1/2 STAAR study can be used for accelerated approval, with eGFR slope at 52 weeks as a key factor. The full data needed for accelerated approval is expected to be ready by the first half of 2025. This new approach could lead to a Biologics License Application (BLA) submission in the second half of 2025, much earlier than previously anticipated, and may eliminate the need for another study to prove effectiveness.
After reviewing the positive safety and efficacy data from the Phase 1/2 STAAR study, Sangamo decided to explore alternative pathways for approval. The FDA agreed that using eGFR slope at 52 weeks as an intermediate clinical endpoint could support accelerated approval. They also suggested looking at eGFR slope at 104 weeks to confirm the clinical benefits of the treatment.
This news is significant for Sangamo as it could potentially speed up the approval process for ST-920 and bring it to market sooner. The use of eGFR slope as a clinical endpoint shows the FDA’s willingness to consider innovative approaches to evaluating the effectiveness of new treatments. This development is a positive step forward for patients with Fabry disease who are eagerly awaiting new treatment options.
Overall, Sangamo’s alignment with the FDA for accelerated approval of ST-920 marks an important milestone in the development of this gene therapy product. The company’s efforts to work closely with the FDA and explore new pathways for approval demonstrate their commitment to bringing innovative treatments to patients in need. This news is a positive sign for the future of ST-920 and for the potential impact it could have on the treatment of Fabry disease.