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

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posted on 2023-05-05, 04:29 authored by Yahiya Y. Syed



Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest Yahiya Y. Syed is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability not applicable

Additional information about this Adis Drug Review can be found here


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 a 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 risk of toxic sphingomyelin catabolites build up, infusion-associated reactions and elevated transaminase levels.

© Springer Nature Switzerland AG 2023


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