Isaralgagene Civaparvovec Fast Tracked for Fabry Disease

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Isaralgagene civaparvovec is a recombinant AAV 2/6 vector carrying the cDNA for human α-galactosidase-A.

The Food and Drug Administration (FDA) has granted Fast Track designation to isaralgagene civaparvovec (ST-920) for the treatment of Fabry disease.

Fabry disease is a rare genetic progressive disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient activity of the lysosomal α-galactosidase-A enzyme. This causes a progressive buildup of globotriaosylceramide (Gb3) in blood vessel walls throughout the body. 

Isaralgagene civaparvovec is a liver-tropic recombinant adeno-associated (rAAV) 2/6 vector carrying the cDNA for human α-galactosidase-A. The investigational product delivers a working copy of the GLA gene to the liver through a single intravenous infusion resulting in the production of functional α-galactosidase-A. 

The safety and tolerability of isaralgagene civaparvovec in adults with Fabry disease is currently being investigated in the open-label, dose-ranging phase 1/2 STAAR study (ClinicalTrials.gov Identifier: NCT04046224). The Company recently announced data providing evidence of clinical benefit, with 13 patients exhibiting sustained, elevated expression of alpha-galactosidase A activity for over 2 years.

“This decision from the FDA underscores the potential for ST-920 to address a serious unmet need and serve as a meaningful therapeutic option for patients with Fabry disease,” said Nathalie Dubois-Stringfellow, PhD, Sangamo’s Senior Vice President, Chief Development Officer. “We are highly encouraged by this promising development and look forward to our expected meeting with the FDA on phase 3 trial design in the summer.”

References:

  1. Sangamo Therapeutics receives US FDA Fast Track designation for isaralgagene civaparvovec for the treatment of Fabry disease. News release. Sangamo Therapeutics. Accessed May 22, 2023. https://www.businesswire.com/news/home/20230522005108/en/Sangamo-Therapeutics-Receives-U.S.-FDA-Fast-Track-Designation-for-Isaralgagene-Civaparvovec-for-the-Treatment-of-Fabry-Disease.