PTC Therapeutics has completed its previously announced agreement with Sangamo Therapeutics to acquire ST-920 (isaralgagene civaparvovec), a one-time AAV gene therapy candidate being developed for Fabry disease.

PTC plans to complete a rolling Biologics License Application (BLA) submission to the US Food and Drug Administration (FDA) in the fourth quarter of 2026, seeking accelerated approval for ST-920.

ST-920 is designed to provide long-term production of the alpha-galactosidase A (α-Gal A) enzyme, which is deficient in people with Fabry disease. Clinical studies have shown sustained enzyme production and reductions in globotriaosylceramide (Gb3) levels, along with clinical benefits and a potential reduction in the need for long-term enzyme replacement therapy (ERT).

The therapy has received Orphan Drug, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. In Europe, it has been granted Orphan Medicinal Product designation and PRIME eligibility by the European Medicines Agency. It has also received Innovative Licensing and Access Pathway designation from the UK Medicines and Healthcare products Regulatory Agency.

 

Source: prnewswire.com

Report Abuse