Sanofi's Rare Disease Drug Xenpozyme Scores FDA Approval

Vandana Singh

Thu, Sep 1, 2022, 4:51 AM 1 min read

Soon after the European Commission's (EC) approval, Sanofi SA (NASDAQ: SNY) scooped up the FDA's OK for the first approved therapy to treat symptoms not related to the central nervous system in patients with acid sphingomyelinase deficiency (ASMD).
The indication includes a range of ailments under the umbrella of Niemann-Pick disease.
Sanofi now has a rare pediatric disease priority review voucher, making it Sanofi's third PRV. The disease afflicts 1 in every 250,000 people.
There are fewer than 120 patients diagnosed with the disease in the US.
The drug replaces a missing enzyme that the body needs to break down a lipid called sphingomyelin.
The lipid accumulates over time in areas like the spleen or lung and liver, spurring swollen, painful abdomens and triggering problems with breathing and eating.
In a small trial, the drug reduced spleen volume by 38.9% for the drug arm, compared to only 0.5% in the control group. The drug showed 23.9% relative improvement in lung function, compared to 3% on placebo.
Japan was the first country to approve the drug back in March under their accelerated SAKIGAKE designation for pioneering drugs.
Price Action: SNY shares are down 0.56% at $40.80 during the premarket session on the last check Thursday.

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