Shares of French pharma major Sanofi (Euronext: SAN) were up more than 2% at 95.05 euros by late morning, as it revealed 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.
Historically known as Niemann-Pick disease types A, A/B, and B, ASMD is a genetically-based, progressive, and potentially life-threatening disease.
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