Highlights 

  • FDA accepts Sangamo’s request for a rolling BLA for its Fabry gene therapy candidate. 
  • Phase 1/2 results indicate treatment potential with multi-organ clinical benefits. 
  • eGFR slope at 52 weeks will serve as the primary basis for approval. 
  • Candidate holds multiple expedited designations in the U.S. and Europe. 
  • Sangamo to begin rolling submission later in Q4 2025 under the accelerated pathway. 

Sangamo Therapeutics (NASDAQ:SGMO) announced that the U.S.Food and Drug Administrationhas accepted its request for a rollingBiologics License Applicationsubmission for isaralgagene civaparvovec, the company's investigational gene therapy for adults with Fabry disease. A rolling submission allows the FDA to review sections of the application as they are completed, offering an earlier assessment timeline. 

Fabry disease is a rare lysosomal storage disorder driven by mutations in the GLA gene, with progressive impact across organs including kidneys, heart, nerves, skin and the gastrointestinal system. Current therapies generally require lifelong administration, underscoring the need for a transformative, longer-lasting approach. 

Clinical Data Supports One-Time, Durable Treatment Potential 

The FDA’s acceptance was guided by encouraging data from Sangamo’s Phase 1/2 STAAR study. Results showed that a single administration of isaralgagene civaparvovec produced favorable multi-organ clinical benefits that surpass outcomes from standard therapies. 

A key metric in the study was the mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks, an indicator of kidney function central to Fabry disease progression. Sangamo reported a positive eGFR slope across all treated patients, a milestone that the FDA has agreed to use as the primary endpoint for approval consideration. 

This alignment reduces regulatory uncertainty and positions the therapy as a potential first-in-class, one-time genetic treatment option for Fabry patients, addressing the disorder at its root. 

Multiple Global Regulatory Designations Strengthen Development Path 

Isaralgagene civaparvovec has received numerous regulatory incentives reflecting its therapeutic promise. In the U.S., the therapy holds Orphan Drug, Fast Track, and RMAT designations. Internationally, it has secured Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency, and recognition under the Innovative Licensing and Access Pathway in the U.K. 

Sangamo plans to initiate the rolling BLA submission later in Q4 2025 under the accelerated approval pathway. With these designations and regulatory momentum, the company is positioned to advance its gene therapy candidate through a streamlined pathway. 

Conclusion 

Sangamo Therapeutics’ progress with its Fabry gene therapy marks a meaningful step in advancing genetic medicine for rare diseases. The FDA’s acceptance of a rolling BLA, coupled with positive clinical indicators and multiple regulatory designations, sets the stage for a potentially significant therapeutic milestone. As the company prepares to begin its submission process, the Fabry disease community awaits what may become a landmark one-time treatment option. 

Sangamo Therapeutics’ shares closed at USD 0.42, marking a 9.97% increase from the prior session. 

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