Sanofi's Rare Disease Drug Xenpozyme Scores FDA Approval

Soon after the European Commission's (EC) approval, Sanofi SA 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.