Introduction and marketing status of olipudase alfa-rpcp

Olipudase (common name: olipudase alfa-rpcp) is developed by Genzyme, a subsidiary of Sanofi (Sanofi) An innovative enzyme replacement therapy for the treatment of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick diseaseB type. This disease is a rare genetic metabolic disease. The patient lacks acid sphingomyelinase, which causes abnormal accumulation of lipids in the liver, spleen, lungs and other organs, thereby causing organ dysfunction. Olipadase helps break down accumulated lipids by supplementing the missing enzyme activity, significantly improving the patient's clinical symptoms.

On 20228month31, olipadaase was approved by the US FDA for marketing, becoming the first enzyme replacement therapy for the treatment of ASMD. This medication is indicated for the treatment of non-CNS symptoms in adult and pediatric patients. In March 2022, Olipadase was approved for marketing by the Japanese Ministry of Health, Labor and Welfare for the treatment of non-central nervous system symptoms of ASMD. In June 2022, the European Medicines Agency (EMA) approved olipadase for the treatment of non-central nervous system symptoms of ASMD.

Olipadase has not yet received marketing approval from the National Medical Products Administration of China (NMPA).

Olipadase, as the world's first enzyme replacement therapy for the treatment of ASMD, has been approved for marketing in the United States, Japan and the European Union, providing a new treatment option for ASMD patients. Although it has not yet received formal marketing approval in China, patients can still get treatment with the drug through Hainan Boao Lecheng's first-in-first-out policy. As global attention to rare diseases increases, it is expected that olipadaase will be approved in more countries and regions, benefiting more ASMD patients.

Reference materials:https://www.xenpozyme.com/