Onasemnogene abeparvovec, marketed under the brand name Zolgensma®, is a valuable treatment option for pediatric patients with spinal muscular atrophy (SMA), according to a systematic review of the literature and meta-analysis published in the Journal of Paediatrics and Child Health.

Onasemnogene abeparvovec is a disease-modifying treatment for SMA indicated for patients under the age of 2 years with all types of SMA. It is a gene therapy that inserts a fully functional copy of the SMN1 gene, which is mutated in SMA, into the body.

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There are few randomized controlled trials supporting the safety and efficacy of onasemnogene abeparvovec. 

In the present study, a team of researchers led by Yuyi Chen from the Department of Pediatric Neurology, Maternal and Child Health Hospital of Guangxi, China, conducted a literature search up to April 2022 and identified 10 studies of onasemnogene abeparvovec that enrolled 250 patients with SMA.

The results of these studies showed that the motor abilities of children with SMA treated with onasemnogene abeparvovec significantly improved in both the short and long term. 

Common adverse events associated with onasemnogene abeparvovec treatment were pyrexia, vomiting, thrombocytopenia, and high levels of aminotransferases, the latter 2 more commonly affecting patients over the age of 8 months.

“In conclusion, despite the paucity of randomized control trial data and low quality of evidence to establish safety and efficacy of onasemnogene abeparvovec treatment, this meta-analysis verifies this is a valuable treatment option for SMA patients,” the authors of the study wrote.

SMA is a rare neurological disease caused by biallelic mutations in the SMN1 gene, which codes for the SMN protein that is essential for the survival of motor neurons. SMA is classified into 4 main types based on the age of onset and severity of symptoms.

Reference

Yang D, Ruan Y, Chen Y. Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: a systematic review and meta-analysis. J Paediatr Child Health. Published online February 1, 2023. doi:10.1111/jpc.16340

Gastroenterologists Neurologists Novartis Gene Therapies Onasemnogene abeparvovec Orthopedists Pediatricians Physical / Occupational Therapists Pulmonologists Zolgensma