Sangamo shares jump as FDA clears faster gene therapy pathway

Shares in Sangamo Therapeutics (Nasdaq: SGMO) surged around 40% on Tuesday, following the announcement that the US Food and Drug Administration has granted the company a faster path to approval for its gene therapy ST-920 (isaralgagene civaparvovec) for Fabry disease.

The FDA's decision, based on data from an ongoing Phase I/II study, means that Sangamo can submit a Biologics License Application as early as 2025, eliminating the need for an additional registrational trial and speeding up approval by approximately three years.

Positive Phase I/II results

The FDA's agreement allows Sangamo to use one-year data from its Phase I/II STAAR study to support accelerated approval, with a focus on kidney function as measured by the estimated glomerular filtration rate (eGFR) slope.

This metric, which tracks kidney health, will serve as the primary basis for approval under the Accelerated Approval program.

Chief executive Sandy Macrae said the firm now has "a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated.” Sangamo expects to have the full dataset ready by mid-2025.

Turning point

This FDA development marks a potential turning point for Sangamo, which has faced several setbacks in recent years.

This has included the termination of collaborations with Biogen (Nasdaq: BIIB), Sanofi (Euronext: SAN), and Novartis (NOVN: VX). Last year, the firm closed its California headquarters and reduced its workforce by 40% to cut costs.

However, it recently gained momentum through a deal with Roche’s (ROG: SIX) Genentech unit, worth nearly $2 billion, for its zinc finger gene editing technologies.

Sangamo's collaboration with Pfizer (NYSE: PFE) on a hemophilia A gene therapy has also posted positive Phase III results, lifting sentiment around the firm’s prospects.