Abstract

A COMPREHENSIVE REVIEW ON THE DRUG PROFILE OF ZOLGENSMA

Dr. M. Govardhan*, Dupati Maha Aishwarya and E. Sumaanjali

ABSTRACT

This review offers an in-depth look at Onasemnogene abeparvovec (Zolgensma®), a gene therapy approved for the treatment of Spinal Muscular Atrophy (SMA). Zolgensma® is delivered as a single intravenous infusion and uses an Adeno-Associated Virus Serotype 9 (AAV9) vector to introduce a functional copy of the human survival motor neuron (SMN) gene into motor neurons. The SMN1 gene is responsible for producing the survival motor neuron protein, which is essential for the health and function of motor neurons. Clinical trials have shown that Zolgensma® significantly improves event-free survival, motor function, and milestones related to motor development in SMA patients. These improvements are sustained for the long term, with some benefits observed for up to five years. The therapy has also been shown to help children with pre-symptomatic SMA achieve motor milestones appropriate for their age, indicating the critical importance of early intervention. While Zolgensma® is generally well tolerated, a known side effect is liver toxicity, which can be managed through the use of preventative steroids like prednisolone. In conclusion, Zolgensma® stands out as a leading treatment option for SMA, particularly when administered early in the course of the disease.

Keywords: Onasemnogene abeparvovec (Zolgensma), Spinal Muscular Atrophy (SMA), Survival Motor Neuron (SMN), Adeno-Associated Virus Serotype 9 (AAV9).