In late May 2023, clinical-stage biopharmaceutical company Sangamo Therapeutics, Inc. (“Sangamo”) shared that its product isaralgagene civaparvovc (ST-920) was granted Fast Track Designation by the FDA. This designation is designed to advance the drug development process by expediting development and review within the rare disease space. It comes with benefits such as more frequent FDA communication and eligibility for Priority Review and Accelerated Approval. Isaralgagene civaparvovc is being developed for people living with Fabry disease.
The Need for Drug Development in the Fabry Disease Space
Fabry disease is a rare genetic lysosomal storage disorder. The genetic mutations cause deficient alpha galactosidase-A, an enzyme that normally breaks down lipids (fats) called globotriaosylceramide. These lipids build up in cells since the body can’t process them. Over time, this causes a number of symptoms and issues that can be life-threatening. Symptoms may include recurrent pain in the hands and feet, hypohidrosis (lowered ability to sweat), gastrointestinal issues, hearing loss, tinnitus, corneal opacity, and red spots on the skin.
Right now, there are limited disease-specific treatments for Fabry disease; the first therapy for adults was only recently approved. There are no disease-specific treatments for children with this condition. Therefore, new therapeutic advances could significantly change patient lives.
Sangamo is evaluating isaralgagene civaparvovc, a recombinant adeno-associated viral vector gene therapy that delivers a working copy of GLA to the body, in the Phase 1/2 STAAR trial. So far, 20 patints have received treatment. By the end of the study, Sangamo hopes to have a better idea of how safe and effective isaralgagene civaparvovc is.
Preliminary study data was presented earlier this year at the WORLDSymposium. This early data suggests that isaralgagene civaparvovc was able to improve kidney health. It also heightened alpha galactosidase-A in a majority of patients, and these raised levels were durable over time.
