Onasemnogene abeparvovec for treating spinal muscular atrophy

Published on 16 September 2025
Last Updated on 16 September 2025

Guidance Recommendations

Onasemnogene abeparvovec is not recommended for inclusion on the MOH Cell, Tissue and Gene Therapy Product List for the treatment of spinal muscular atrophy (SMA) in children under 2 years of age who have Type 1 SMA or up to three copies of the survival motor neuron 2 gene.

This is because the current available evidence is insufficient to show that onasemnogene abeparvovec provides superior long-term efficacy compared to risdiplam, the subsidised disease-modifying treatment for SMA.

Given the current uncertainty in its superiority to risdiplam and its higher treatment cost, it is deemed that the cost-effectiveness of onasemnogene abeparvovec is unfavourable when compared to risdiplam at this time.

Onasemnogene abeparvovec for treating spinal muscular atrophy (Published 16 Sep 2025)

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