Olipudase alfa's long-term impact in ASMD shows promise

Positive results from long-term, open-label extension studies demonstrated that olipudase alfa provided sustained improvement in lung function and reduction of spleen and liver volumes in adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD).

French drugmaker Sanofi’s (Euronext: SAN) investigational enzyme replacement therapy is designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin. It is hoped that it can provide a treatment a rare, progressive, and potentially life-threatening disease with no approved treatments that has previously been known as Niemann-Pick disease type A and type B.

"Currently, patients living with this extremely rare disease have no treatment options"The new data presented consist of 6.5-year outcomes for five adult patients with ASMD and two-year outcomes in 20 pediatric patients, as well as the open-label extension from the Phase III ASCEND trial in adults.