The U.S. FDA has provided a clear regulatory pathway to accelerated approval for Sangamo Therapeutics’ gene therapy product candidate for the treatment of Fabry disease, speeding up the timeline for potential approval by approximately three years.
According to Sangamo, during a recent Type B interaction with the FDA regarding isaralgagene civaparvovec (ST-920), the agency agreed that data from the ongoing phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program.
The complete dataset to support the accelerated pathway will be available in the first half of 2025, teeing up a potential BLA submission in the second half of 2025 — three years ahead of previous estimates. This would also mean that an additional registrational study to establish clinical efficacy is not needed.
It’s great news for Sangamo considering that a year ago, in an effort to reduce operating expenses, the company cut its U.S workforce by 40% and streamlined its pipeline. At the time, Sangamo had made the decision todefer additional investments in the phase 3 planning for its Fabry disease asset until a collaboration partner or phase 3 trial funding could be secured.
Fabry disease is a rare lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity. Sangamo’s Isaralgagene civaparvovec is a liver-tropic rAAV 2/6 vector carrying the cDNA for human α-Gal A that is delivered through a single dose IV infusion. It aims to deliver a working copy of the GLA gene to the liver so that liver cells can start producing functional α-Gal A.