SEARCHNARRATIVE_REVIEWpediatrics, internal medicine

narrative_review·pediatrics, internal medicine, pulmonology, clinical trial, preclinical research·PMC10361862

Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use

Clinical Drug Investigation · 1 author, 1 centre

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The review notes that patient-reported outcomes did not show significant between-group differences in the ASCEND trial.

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The review notes that patient-reported outcomes did not show significant between-group differences in the ASCEND trial.

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narrative_review

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pediatrics

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AI pipeline

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100% · A

ORIGINAL PAPER

PMC10361862

Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use

Clinical Drug Investigation

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ABSTRACT

Abstract Olipudase alfa (Xenpozyme™) is an intravenously administered acid sphingomyelinase enzyme replacement therapy indicated to treat non-CNS manifestations of acid sphingomyelinase deficiency (ASMD) in adult and paediatric patients. It is the first and currently the only disease-modifying treatment for ASMD. Olipudase alfa treatment improves hepatosplenomegaly, lung function and platelet counts, along with multiple other pathological features of ASMD in adult and paediatric patients with ASMD. These benefits are sustained through at least 24 months of treatment. Olipudase alfa is generally well tolerated; infusion-associated reactions (mostly mild) were the most common treatment-related adverse events. Other warnings and precautions associated with its use include risks of hypersensitivity reactions (including anaphylaxis) and elevated transaminase levels seen in clinical trials, and foetal malformation based on animal studies. All these risks are generally manageable. A gradual dose escalation of olipudase alfa, followed by a maintenance phase, is required to reduce the risks of toxic sphingomyelin catabolites build up, infusion-associated reactions and transient transaminase elevations. Supplementary Information The online version contains supplementary material available at 10.1007/s40261-023-01270-x.

CITATION EXPORT

Yahiya Y Syed. Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use. Clinical Drug Investigation. (2023). https://doi.org/10.1007/s40261-023-01270-x

AUTHORS

1 · 1 CENTRES

Yahiya Y Syed

1Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754 New Zealand