EMA Accepts MAA for Sanofi’s Rare Disease Therapy

The European Medicines Agency (EMA) has accepted Sanofi’s marketing authorization application (MAA) for olipudase alfa, an investigational enzyme replacement therapy being evaluated for the treatment of acid sphingomyelinase deficiency (ASMD). The MAA will be reviewed under an accelerated assessment procedure, and a European regulatory decision is anticipated in the second half of 2022, Sanofi announced in a Dec. 6, 2021, press release.

ASMD is historically referred to as Niemann-Pick disease (NPD) type A and type B. ASMD is a rare, progressive, and potentially life-threatening disease for which no treatments are currently approved, Sanofi stated in the press release. ASMD is estimated to affect approximately 2000 patients in the United States, Europe, and Japan. If approved, olipudase alfa will become the first and only therapy for treating this disease, according to Sanofi.

The MAA is based on positive results from two separate clinical trials that evaluated olipudase alfa in adult and pediatric patients with non-central nervous system manifestations of ASMD type A/B and ASMD type B. Olipudase alfa has received priority medicines designation from the EMA, breakthrough therapy designation from FDA, and Sakigake designation from Japanese regulatory authorities. Sanofi filed a Japanese new drug application for olipudase alfa in September 2021.

“Today’s milestone has been decades in the making, and our gratitude goes to the ASMD community who has stood by us with endless patience while olipudase alfa advanced through clinical development,” said Alaa Hamed, global head of Medical Affairs, Rare Diseases, Sanofi, in the press release. “Olipudase alfa represents the kind of potentially life-changing innovation that is possible when industry, medical professionals and the patient community work together toward a common goal.”

Source: Sanofi