Sanofi: Xenpozyme Gets Marketing Authorization In Japan For ASMD

French drugmaker Sanofi (SNYNF,SNY) announced Monday that the Japanese Ministry of Health, Labor, and Welfare or MHLW has granted marketing authorization for Xenpozyme (olipudase alfa) to treat acid sphingomyelinase deficiency or ASMD.

Xenpozyme is approved for the treatment of adult and pediatric patients with non-central nervous system or non-CNS manifestations of ASMD, a rare, progressive, and potentially life-threatening genetic disease.

Xenpozyme is currently the first and only approved treatment for ASMD and represents Sanofi’s first therapy to be approved under Japanese government’s regulatory fast-track SAKIGAKE designation.

The approval in Japan was based on positive results from two separate clinical trials, ASCEND and ASCEND-Peds, in children and adults demonstrating improvement in lung function, as measured by Dlco, and reduction of spleen and liver volumes.

The data showed that Xenpozyme provided improvement in lung function, as measured by diffusing capacity of the lung for carbon monoxide, or Dlco, and reduction of spleen and liver volumes, with a well-tolerated safety profile in adults and children with ASMD.

These data were presented at the American Society of Human Genetics (ASHG) 2020 Virtual Meeting.

Outside of Japan, olipudase alfa is being evaluated by regulatory authorities around the world. The U.S. Food and Drug Administration accepted a Biologics License Application for olipudase alfa for Priority Review, with a decision expected early Q3 2022.

The European Medicines Agency has awarded olipudase alfa the PRIority MEdicines or PRIME designation, and a decision is anticipated in the second half of 2022.

In Paris, Sanofi shares were trading at 94.38 euros, up 1.35 percent.

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