Enzyme replacement for genetic disease

Thursday 1 September 2022


An enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a rare genetic disease that can cause premature death, has cleared the US Food and Drug Administration making it the first medicine to treat symptoms of the disease that are not related to the central nervous system. The FDA approval of Xenpozyme (olipudase alfa) was granted to Genzyme, a division of Sanofi SA.