Olipudase alfa meets endpoints in ASMD study

Olipudase alfa, an investigational recombinant human acid sphingomyelinase, demonstrated positive results in two separate clinical trials evaluating olipudase alfa for the treatment of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, says French pharma major Sanofi (Euronext: SAN).

Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD. No treatments are currently approved for ASMD, also known as Niemann-Pick disease type B. The product received Breakthrough Therapy designation from the US regulator in 2015

“These significant results for olipudase alfa mark a major scientific advancement for ASMD and an important step toward providing a potential therapy for adult and pediatric patients who currently have no approved treatment options for this devastating disease,” said Dr John Reed, global head of R&D at Sanofi.