Onasemnogene abeparvovec

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA),[6][7] a disease causing muscle function loss in children.

Onasemnogene abeparvovec, a biologic drug utilizing AAV9 virus capsids containing an SMN1 transgene, is administered to motor neurons, boosting SMN protein levels.

Onasemnogene abeparvovec's price is high, earning it the title of the world's most expensive medication at the time of commercial approval.

[11][12] Onasemnogene abeparvovec has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the SMN1 gene on chromosome 5q[7] and diagnosed predominantly in young children that causes progressive loss of muscle function and frequently death.

[13] In the United States, onasemnogene abeparvovec is indicated for the treatment of people less than two years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

[6] The treatment is approved in the United States and certain other countries for use in children with spinal muscular atrophy up to the age of two, including at the presymptomatic stage of the disease.

[citation needed] Onasemnogene abeparvovec, developed by the US biotechnology startup AveXis, which was acquired by Novartis in 2018,[17] is based on research conducted at the Institut de Myologie in France.

[14] Additionally, the FDA awarded the manufacturer a rare pediatric disease priority review voucher and approved onasemnogene abeparvovec for AveXis Inc.[14] In June 2015, the European Commission granted orphan designation to the drug.

[10] The Central Social Insurance Medical Council, responsible for approving the universal drug fee schedule in Japan, has negotiated the price down to ¥167,077,222 (approx.

[37] In October 2019, the company admitted to not having informed the FDA and the European Medicines Agency (EMA) for seven months about toxic effects of the intravenous formulation observed in laboratory animals.