Sanofi gets Japenese nod for Xenpozyme to treat acid sphingomyelinase deficiency
Xenpozyme is a recombinant human acid sphingomyelinase enzyme developed to replace deficient or defective acid sphingomyelinase (ASM);
Paris: Drugmaker, Sanofi, today announced that the Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Xenpozyme (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease.
Xenpozyme is currently the only approved treatment for ASMD and represents Sanofi's first therapy to be approved under the SAKIGAKE (or "pioneer") designation, which is the Japanese government's regulatory fast-track pathway to promote research and development of innovative new medical products addressing urgent unmet medical needs.
"The approval of Xenpozyme is a watershed moment for ASMD patients and their families, representing 20 years of research and the shared efforts of advocacy partners, clinicians, and patients. As the world's first medicine approved for ASMD, Xenpozyme offers a potentially transformative option for this historically neglected community. We are proud of this achievement and grateful that Japan's PDMA has recognized the significance of the unmet need that Xenpozyme addresses with the Sakigake designation. At Sanofi, we are working with health authorities globally, including the EU where olipudase alfa has PRIME designation and in the USA where this enzyme replacement therapy has Breakthrough designation, to rush this important medicine to ASMD patients around the world," said John Reed, M.D., Ph.D, Executive Vice President, Global Head of Research and Development, Sanofi.
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