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Sanofi announced that Xenpozyme (olipudase alfa-rpcp) won FDA approval for adult and pediatric patients with the rare genetic disease acid sphingomyelinase deficiency (ASMD), often referred to as Niemann-Pick disease.

In its announcement, the FDA noted that Xenpozyme’s approval is for ASMD symptoms unrelated to the central nervous system. Xenpozyme is the first and only disease-specific drug approved by the FDA for ASMD. The approval was based on promising findings from the Phase II/III ASCEND trial, which enrolled 31 adult patients with ASMD type B or type A/B. Participants were randomly assigned to receive Xenpozyme or a saline placebo through 52 weeks of treatment, after which ASCEND assessed lung function and spleen and liver size. Twelve patients treated with Xenpozyme saw a 23.9% relative improvement in lung function compared to the 3% improvement in placebo comparators. Spleen and liver volume likewise dropped by 38.9% and 26.5%, respectively, after 52 weeks of Xenpozyme. Those in the placebo arm saw spleen volume increase by 0.5% and liver volume decrease by 1.8%.

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