Date: 2015-02-12
Type of information: Presentation of results at a congress
phase: 1b
Announcement: presentation of results at the Lysosomal Disease Network’s WORLD Symposium 2015 in Orlando
Company: Genzyme (USA - MA), a Sanofi company (France)
Product: olipudase alfa
Action
mechanism: enzyme replacement therapy. Olipudase alfa is a recombinant human acid sphingomyelinase. Traditionally called Niemann-Pick Disease types A and B (NPD A and NPD B), acid sphingomyelinase deficiency (ASMD) is one of a group of lysosomal storage diseases that affect the metabolism and that are caused by genetic mutations. ASMD is caused by the deficiency of a specific enzyme, acid sphingomyelinase (ASM). This enzyme is found in special compartments within cells called lysosomes and is required to metabolize a lipid called sphingomyelin. If ASM is absent or not functioning properly, sphingomyelin cannot be metabolized properly and is accumulated within the cell, eventually causing cell death and the malfunction of major organ systems. Niemann-Pick A and Niemann-Pick B are both caused by the same enzymatic deficiency and there is growing evidence that the two forms represent opposite ends of a continuum.
Disease: Niemann-Pick Disease type B
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: * On February 12, 2015, Genzyme, a Sanofi company, presented data from its Phase 1b clinical study at the Lysosomal Disease Network’s WORLD Symposium 2015 in Orlando, detailing the investigational use of enzyme replacement therapy in the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type B), a lysosomal storage disease caused by genetic mutations that affect the metabolism of sphingomyelin. The Genzyme study evaluated the tolerability and safety of olipudase alfa (recombinant human acid sphingomyelinase) in five adult patients with ASMD. Melissa P. Wasserstein, MD, Director of the Program for Inherited Metabolic Diseases; Medical Director of the International Center for Types A and B Niemann Pick Disease, Mount Sinai School of Medicine, presented: An open-label, multicenter, ascending-repeat-dose study of the tolerability and safety of recombinant human acid sphingomyelinase (rhASM) in patients with ASM deficiency (ASMD). In the trial, each patient received a starting dose of intravenous olipudase alfa at 0.1 mg/kg and escalated dosing every two weeks according to a predetermined schedule up to 3 mg/kg or their maximum tolerated dose. The secondary objective was to study the pharmacokinetics, pharmacodynamics, and exploratory efficacy of olipudase alfa administered every two weeks for 26 weeks. The study findings showed that the dose escalation regimen was well tolerated, with all patients reaching the maximum dose of 3 mg/kg. No serious or severe adverse events or deaths were reported. The data presented on the repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa support its continued development for the investigational use in non-neurological manifestations of ASMD. All five patients are participating in the Long-Term Study and will continue on therapy. Genzyme now plans to begin enrolling patients in a Phase 2/3 program for Niemann-Pick Type B in 2015.
