Sanofi subsidiary Genzyme has started Phase I/II clinical trial to assess the efficacy of its investigational therapy olipudase alfa in paediatric patients.
Olipudase alfa is an enzyme replacement therapy that is being studied to treat non-neurological manifestations of acid sphingomyelinase deficiency (ASMD) or otherwise called Niemann-Pick disease type B.
ASMD is a serious and life-threatening disorder, which is induced by insufficient activity of the enzyme acid sphingomyelinase (ASM) resulting in toxic accumulation of sphingomyelin and there are currently no approved treatments options to treat this disease.
Sanofi Genzyme R&D Centre head Dr Jim Burns said: "With start of clinical studies both in pediatric and adult patients just a few months apart, we are confirming our commitment to the Niemann Pick disease patient community to advance the development of this novel therapy as quickly as possible and for both patient populations.
"We are very grateful to the patients for their engagement and their support in advancing this exciting programme."
The Phase I/II study is a multi-national, multi-centre, open label and ascending dose trial to assess the safety, tolerability and pharmacokinetics of olipudase alfa administered intravenously once every two weeks for 52 weeks in paediatric patients with ASMD.
In the trial, 12 paediatric patients will be enrolled into three age cohorts, including an adolescent cohort, child cohort and an infant/early child cohort.
According to the firm, the primary objective of the study is to evaluate the safety and tolerability of olipudase alfa and patients will have the option to enrol into an extension study, upon completion of the 52-week trial.
Genzyme received breakthrough therapy designation from the US Food and Drug Administration (FDA) for olipudase alfa.
Image: The entrance to Genzyme building at 500 Kendall Square in Cambridge, Massachusetts. Photo: courtesy of Tim Pierce.