Sanofi announced that the U.S. FDA has granted Breakthrough Therapy designation to its investigational drug venglustat for the treatment of neurological symptoms in type 3 Gaucher disease, a rare genetic disorder with no approved therapies targeting central nervous system involvement.

The designation is based on Phase 3 trial data showing statistically significant improvements in neurological function compared to standard enzyme replacement therapy, with a favorable safety profile. Venglustat is designed to cross the blood-brain barrier and reduce the accumulation of harmful molecules driving disease progression.

The status is expected to accelerate development and regulatory review, with Sanofi planning to pursue global filings for the treatment in 2026, addressing a significant unmet medical need in this patient population.