Sangamo Therapeutics Secures Accelerated Approval Pathway for Gene Therapy in Fabry Disease

FDA Approves Accelerated Pathway

The FDA’s decision is based on promising data from the ongoing Phase 1/2 STAAR study, which has demonstrated significant improvements in kidney function among patients. The agency has agreed that the estimated glomerular filtration rate (eGFR) slope at 52 weeks can be used as the primary endpoint for approval, avoiding the need for an additional registrational study.

Potential for Earlier Approval

The full dataset to support this Accelerated Approval is expected in the first half of 2025. Sangamo has already initiated preparations for the BLA submission while engaging in partnership discussions to advance the development of this gene therapy.

About Fabry Disease

Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene. These mutations result in the deficiency of an enzyme called alpha-galactosidase A, leading to the buildup of a fatty substance known as globotriaosylceramide (Gb3). Over time, this accumulation damages vital organs, including the kidneys, heart, and nervous system, causing a wide range of symptoms such as kidney disease, heart failure, and neuropathic pain. If untreated, Fabry disease can lead to life-threatening complications, emphasizing the need for effective therapies.

Addressing Unmet Medical Needs

Sangamo’s gene therapy offers new hope for Fabry disease patients by targeting the underlying cause of the disease. With the Accelerated Approval pathway, this treatment could reach patients significantly sooner, providing a much-needed option for improving their quality of life and addressing the serious health risks posed by the disease.

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