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 individuals with type B disease receive treatment for dyslipidemia, nutritional consultations and, occasionally, blood transfusions.
A number of clinical trials are investigating the potential of enzyme replacement therapy in treating adults with the disease.
Sanofi notes that if olipudase alfa is approved, it would become the first therapy for ASDM.
Filed Under: clinical trials, Drug Discovery