Sanofi's Enzyme Replacement Therapy Xenpozyme Receives First Global Approval in Japan for Acid Sphingomyelinase Deficiency

Today, Sanofi announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved the company's innovative enzyme replacement therapy Xenpozyme (olipudase alfa) for marketing, used to treat non-central nervous system symptoms in adult and pediatric patients with acid sphingomyelinase deficiency (ASMD). This is a rare, potentially fatal genetic disorder. The press release noted that Xenpozyme is the first approved treatment for ASMD.

Xenpozyme is a recombinant human acid sphingomyelinase protein designed to replace the missing or defective acid sphingomyelinase (ASM). It is responsible for breaking down sphingomyelin. Due to mutations in the gene encoding this metabolic enzyme, patients with ASMD experience an accumulation of sphingomyelin in their cells, leading to damage in organs such as the lungs, spleen, and liver, which may result in premature death.

The approval of Xenpozyme in Japan was based on clinical trials conducted in adult and pediatric patients. The trial results showed that Xenpozyme improved lung function in patients, reduced spleen and liver volume, and demonstrated good safety and tolerability in both adult and pediatric patients.

Outside of Japan, the Biologics License Application (BLA) for olipudase alfa has been granted Priority Review by the U.S. FDA, with a regulatory decision expected in the third quarter of this year. It has previously received Breakthrough Therapy Designation from the U.S. FDA.

References:

[1] Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency. Retrieved March 28, 2022, from https://www.globenewswire.com/news-release/2022/03/28/2410648/0/en/Press-Release-Xenpozyme-olipudase-alfa-approved-in-Japan-first-and-only-approved-therapy-indicated-to-treat-acid-sphingomyelinase-deficiency.html

Original Title:Express | World's First! Sanofi's Innovative Enzyme Replacement Therapy Approved in Japan

Note: The original text has been abridged.

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