EMA has recommended granting a marketing authorisation in the European Union (EU) for Xenpozyme (olipudase alfa), a therapy for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disease. Xenpozyme is indicated for use in children and adults of all ages with type A/B or type B ASMD.
Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder. It belongs to the larger family of metabolic disorders called ‘lysosomal storage diseases’, in which fats build up within the parts of the body’s cells that break down nutrients and other materials. This affects the way cells work and causes them to die, affecting normal functioning of tissues and organs. ASMD is seriously debilitating and life threatening since the build-up of fatty substances can cause brain damage and swelling of organs such as liver and spleen…
