The US Food and Drug Administration (FDA) has cleared Sanofi’s Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD).

According to Sanofi, Xenpozyme (olipudase alfa-rpcp) is the first and only approved therapy for the treatment of ASMD in adult and paediatric patients.

ASMD is a rare genetic disease that can cause premature death.

Xenpozyme is an enzyme replacement medication that aids in lowering sphingomyelin buildup in the liver, spleen, and lung.

The approval is based on the data from ASCEND and ASCEND-Peds trials, in which Xenpozyme showed improvement in lung function, platelet count, and reduction of spleen and liver volumes.

A trial with 31 patients who were randomly assigned to receive either Xenpozyme or a placebo showed that the drug is effective for treating ASMD.

The FDA was able to draw the conclusion that Xenpozyme is efficacious since the trial included a placebo comparator and assessed treatment advantages that could be noticed over the course of the study.

Overall, Xenpozyme therapy lowered liver and spleen size and improved lung function.

It has secured the orphan drug classification, which offers rewards to help and promote the development of medications for uncommon disorders.

The FDA granted the sponsor a voucher for a rare paediatric disease priority review as a financial incentive to promote the creation of novel medications and biologics that prevent and treat rare paediatric disorders.

Xenpozyme was given the title of Breakthrough Therapy in the US, which speeds up the development and approval of medications used to treat critical or life-threatening illnesses and disorders.

Xenpozyme was subjected to a Priority Review by the FDA, a process designated for drugs with the potential to significantly improve their ability to treat critical illnesses safely or efficaciously.

The SAKIGAKE designation was given to Xenpozyme in March 2022 in Japan, making it the first olipudase alfa product to receive such a classification globally.

The use of Xenpozyme in Europe was authorised by the European Commission (EC) in June 2022.