[4][5][6][7] The most common side events include infections, infusion-related reactions, or gastrointestinal complaints (disease signs and symptoms in children).
[5] 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.
[5] Acid sphingomyelinase deficiency 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.
[5] Olipudase alfa was approved for medical use in Japan in March 2022,[6] in the European Union in June 2022,[5] and in the United States in August 2022.
[5] Xenpozyme was reviewed under the accelerated assessment program of the European Medicines Agency (EMA).