US FDA Approves Sanofi’s Rare Disease Drug Xenpozyme


In a campaign to develop the first and best-in-class medicines, Sanofi has gained approval from the US Food and Drug Administration (FDA) for Sanofi’s latest rare disease drug, known as Xenpozyme.

In March 2022, the health regulators in Japan signed off on Xenpozyme to treat Niemann-Pick, which is a rare genetic disease that prevents the body from metabolizing fat and also leads to premature death. The same was done by the health officials of Europe months later.

The US FDA said currently, the intravenously infused treatment will be available to adults and children in the United States. It is the very first drug approved by the US FDA for the non-central nervous system (non-CNS) manifestations of the condition, which is also known as acid sphingomyelinase deficiency (ASMD).

Sanofi said that there are fewer than 120 people who are diagnosed with ASMD in the United States, with around two-thirds of the patients being children. This rare condition is caused by a lack of an enzyme, which is needed to break down a complex lipid, known as sphingomyelin that collects in the spleen, liver, brain, and lung.

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According to Sanofi, Xenpozyme is a hydraulic lysomal enzyme replacement therapy that reduces the accumulation of sphingomyelin. This new drug is not expected to change the CNS manifestation of ASMD.