NEW YORK – After a meeting with the US Food and Drug Administration, Sangamo Therapeutics' regulatory path for its Fabry disease gene therapy, isaralgagene civaparvovec (ST-920), has become clearer.
The Richmond, California-based firm said Monday that the FDA has agreed that Sangamo can submit in its biologics license application seeking approval for isaralgagene civaparvovec data from a well-controlled study involving up to 25 male and female patients that doesn't have a control arm. "A head-to-head comparison with enzyme replacement therapy is not part of the proposed study design deemed acceptable by the FDA," Sangamo said in a statement, noting that it will have to provide the agency confirmatory evidence of the treatment's activity. "This approach enables a potentially more rapid, efficient, and cost-effective pathway to BLA submission than originally anticipated."
However, despite having this guidance from the FDA about a potential BLA for isaralgagene civaparvovec, Sangamo said it will not invest in conducting a registrational trial until it secures a commercialization partner, which it is actively trying to do.
Isaralgagene civaparvovec is designed to deliver a functional copy of the GLA gene to the liver to increase production of functional alpha-Gal A, an enzyme needed to metabolize globotriaosylceramide. Patients with Fabry disease, a rare lysosomal storage disorder, have a deficiency of this enzyme.
Sangamo is studying the gene therapy in the Phase I/II STAAR study, from which it recently reported updated data demonstrating isaralgagene civaparvovec's efficacy and safety in 24 patients. The company has finished screening and enrolling patients in this study and hopes to treat all enrolled patients by the first half of this year.
Additionally, the European Medicines Agency has deemed isaralgagene civaparvovec eligible for priority medicines designation, which will provide Sangamo additional support with the development and regulatory planning for the agent. US and European regulators have also already deemed isaralgagene civaparvovec an orphan drug, and it has fast-track and regenerative medicine advance therapy designation from the FDA.