Genzyme nabs breakthrough designation for rare enzyme disorder
- Genzyme has received an FDA priority review designation for olipudase alfa, intended to replace missing enzymes in patients with ASMD—a condition which often lead to severe neurological symptoms.
- ASMD stands for acid sphingomyelinase deficiency, which is also known as Niemann-Pick disease type B.
- Supplementing patients who have ASMD with olipudase alfa allows for the breakdown of sphingomyelin.
Currently, the ability to treat Niemann-Pick disease type B is an unmet medical need. Genzyme's drug is designed to replace the enzyme, which metabolizes sphingomyelin. When sphingomyelin is not processed properly it accumulates in the body leading to numerous problems, including abdominal distention, pain, dysphagia, and numerous CNS symptoms, such as unsteady gait.
Genzyme won Breakthough Therapy designation based on the strength of its clinical trial data from a phase 1b study. Genzyme is now enrolling for the next round of trials, which will include a Phase 1/2 pediatric study and a Phase 2/3 adult study.