The approval comes after more than two decades of research and development by rare disease specialist Genzyme, which Sanofi bought in 2011 for $20.1 billion.
Patients with ASMD suffer from a lack of an enzyme needed to break down a lipid known as sphingomyelin that can build up in cells and affect organ function, leading to debilitating symptoms. Xenpozyme, given by infusion every two weeks, is designed to replace the missing enzyme.
