EMA reviews Sanofi's olipudase alfa for rare ASMD disease

The European Medicines Agency (EMA) has accepted Sanofi's marketing authorization application for olipudase alfa for review under an accelerated assessment procedure. The investigational enzyme replacement therapy is being evaluated for the treatment of acid sphingomyelinase deficiency (ASMD).

ASMD, historically referred to as Niemann-Pick disease types A and B, is a rare, progressive, and potentially life-threatening condition for which no treatments are currently approved. An estimated 2,000 patients across the U.S., Europe, and Japan are affected. If approved, olipudase alfa would become the first and only therapy available for ASMD.

The application is supported by positive results from two clinical trials, ASCEND and ASCEND-Peds, which evaluated olipudase alfa in adult and pediatric patients with non-central nervous system manifestations of ASMD. The EMA's PRIME designation for olipudase alfa signifies its potential to offer a major therapeutic advantage.

Olipudase alfa has also received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and SAKIGAKE designation in Japan, recognizing its potential for seriously ill patients with unmet medical needs. A regulatory decision from the EMA is anticipated in the second half of 2022.

ASMD results from deficient activity of the enzyme acid sphingomyelinase, leading to the accumulation of sphingomyelin within cells, potentially causing cell death and organ dysfunction.