Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Sanofi company Genzyme has dosed first pediatric patient in its Phase I/II clinical trial of olipudase alfa, an enzyme replacement therapy, to treat nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), a serious and life-threatening disorder.
Subscribe to our email newsletter
Olipudase alfa is being evaluated to treat nonneurological manifestations of ASMD, also known as Niemann-Pick disease type B, as opposed to type A, which is characterized by neurological involvement.
ASMD is caused by insufficient activity of the enzyme acid sphingomyelinase (ASM) resulting in toxic accumulation of sphingomyelin.
Currently, there are no approved treatment options for patients with Niemann-Pick disease type B.
The multi-national, multi-center, open label, ascending dose Phase I/II trial will evaluate the safety, tolerability and pharmacokinetics of olipudase alfa administered intravenously once every two weeks for 52 weeks in pediatric patients with ASMD.
A total of 12 pediatric patients will be enrolled into three age groups: an adolescent cohort 12 to <18 years of age; a child group six to <12 years of age and an infant/early child group birth to < six years of age.
The trial’s primary objective is to evaluate the safety and tolerability of olipudase alfa.
Sanofi Genzyme R&D Center head 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."
Following completion of the 52-week trial, patients will have the option to enroll into an extension study.
In the second half of this year, the company intends to begin enrollment in a Phase II/II trial involving adult patients with ASMD.
Usually known as Niemann-Pick disease types A and B (NPD A and NPD B), ASMD is one of a group of lysosomal storage diseases that affect the metabolism and that are caused by genetic mutations.
Image: Genzyme’s Phase I/II trial is designed to evaluate olipudase alfa administered intravenously in pediatric patients with ASMD. Photo: courtesy of Baitong333/ freedigitalphotos.net.