Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.

ABSTRACT:

Background

Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD; Niemann-Pick Disease (NPD) A and B]. This open-label phase 1b study assessed the safety and tolerability of olipudase alfa using within-patient dose escalation to gradually debulk accumulated sphingomyelin and mitigate the rapid production of metabolites, which can be toxic. Secondary objectives were pharmacokinetics, pharmacodynamics, and exploratory efficacy.

Methods

Five adults with nonneuronopathic ASMD (NPD B) received escalating doses (0.1 to 3.0 mg/kg) of olipudase alfa intravenously every 2 weeks for 26 weeks.

Results

All patients successfully reached 3.0mg/kg without serious or severe adve

SUBMITTER: Wasserstein MP 

PROVIDER: S-EPMC4561589 | biostudies-literature | 2015 Sep-Oct

REPOSITORIES: biostudies-literature