Spur Therapeutics Gains FDA Support for Phase 3 Gaucher Gene Therapy Trial

Spur Therapeutics has secured FDA alignment on its Phase 3 trial plans for FLT201, a gene therapy for Gaucher disease type 1. Encouraging results from the Phase 1/2 GALILEO-1 trial demonstrated rapid and sustained reductions in lyso-Gb1, a key biomarker. The FDA's feedback supports the potential for accelerated approval based on lyso-Gb1 reductions, with full approval hinging on hemoglobin maintenance at one year. Spur plans to dose the first patient in late 2025, aiming to offer a groundbreaking one-time treatment option.

CEO Michael Parini emphasized the significance of the decision, stating that FLT201 could set a new standard for Gaucher care by reducing symptoms and treatment burdens. The upcoming Phase 3 study will enroll 40-45 patients worldwide, assessing outcomes such as hemoglobin levels, platelet counts, organ volume, bone health, and overall quality of life. With over 45 trial sites in preparation across multiple countries, Spur is moving quickly to bring this potentially life-changing therapy to patients.