Health Ministry has approved a drug for the treatment of Niemann–Pick disease

The Ministry of Health of the Russian Federation issued a regulatory certificate to the Dutch company Genzyme Europe (Genzyme has been owned by Sanofi since 2011) for the drug Zenpozyme (olipudase alfa), intended for the treatment of a rare genetic disease – Niemann-Pick disease A/B and B types. In 2023, this disease was included in the roster of conditions entitling individuals to receive treatment funded by the “Circle of Kindness” state fund.

Genzyme was an American biotechnology company based in Cambridge, Massachusetts. Since its acquisition in 2011, Genzyme (also known as Genzyme Transgenics Corp or GTC Biotherapeutics) has been a fully owned subsidiary of Sanofi.

Xenpozyme is a medicine for treating patients with acid sphingomyelinase deficiency (ASMD), a genetic condition, historically known as Niemann-Pick disease type A, A/B and B. There are three types of Niemann-Pick disease (A, B and C), with different genetic causes and different symptoms. Xenpozyme is used to treat patients with type A/B or type B. It is intended to treat the symptoms of ASMD that are not related to the brain. Xenpozyme contains the active substance olipudase alfa.

Niemann-Pick disease is rare, and Xenpozyme was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 5 December 2016. Further information on the orphan designation can be found here: ema.europa.eu/medicines/human/orphan-designations/eu-3-01-056.

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