Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced important U.S. and European regulatory updates for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease.

The FDA has agreed in a Type D meeting that data from a single, adequate, and well-controlled study may form the primary basis of approval of a BLA for isaralgagene civaparvovec. The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm. A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA. This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.

Additionally, the EMA has granted PRIME eligibility to isaralgagene civaparvovec. PRIME is a program designed to enhance support for the development of medicines that target an unmet medical need and is intended to optimize development plans and expedite review and approval processes so that these medicines may reach patients as early as possible. Isaralgagene civaparvovec has already received Orphan Medicinal Product designation from the EMA as well as Orphan Drug, Fast Track and RMAT designations from the FDA.

“The U.S. and European regulatory support for ST-920 and the serious unmet medical need in Fabry Disease signal the important role that ST-920 could play in improving the lives of Fabry patients across the globe,” said Nathalie Dubois Stringfellow, Ph.D., Chief Development Officer of Sangamo. “We are thankful for the FDA’s support and alignment on a regulatory pathway that could potentially deliver a new treatment option for Fabry disease patients on an expedited, cost-effective timeline. Similarly, we appreciate the support from the EMA and the opportunity to advance our development plans in Europe. Fabry is a debilitating disease in need of new medicines, and we are grateful that regulatory agencies across geographies recognize this and support our proposed development plans.”

Updated Phase 1/2 STAAR study data showing sustained clinical benefit and a differentiated safety profile across 24 patients were shared at the 20^th Annual WORLDSymposium^TM in San Diego, CA on Wednesday, February 7, 2024. A total of 29 patients have been treated to date in the Phase 1/2 STAAR study. All 13 patients withdrawn from ERT remain off ERT as of February 12, 2024. Screening and enrollment are complete in the study and dosing of the remaining enrolled patients is expected in the first half of 2024. Sangamo is deferring additional investments in planning for a registrational trial until a collaboration partnership is secured.