Sanofi (NSDQ:SNY) has announced that olipudase alfa offered a sustained improvement in patients with acid sphingomyelinase deficiency (ASMD) in investigational long-term follow-up studies.
ASDM is a rare progressive and potentially life-threatening disease with no authorized treatments.
Patients in the trial had improvements in terms of lung function and reduced spleen and liver volumes.
The research focused on non-central nervous system (non-CNS) manifestations of ASDM.
A sphingomyelin-phosphodiesterase replacement, olipudase alfa, has been the subject of seven clinical trials to date. To date, treatment of ASDM, also known as ASM-deficient Niemann-Pick disease, depends on whether patients have a manifestation known as Niemann-Pick disease type A (NPD-A) or Niemann-Pick disease type B (NPD-B).
Patients with the first type may receive physical and occupational therapy and nutritional consultations.
Some individual…