Novartis submitted its spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) for marketing approval in China on 2 August, following its inclusion in the National Medical Products Administration's (NMPA) priority review pathway. The one-time intravenous treatment, priced at USD 2.1 million in the US, targets SMA patients aged six months and older with bi-allelic SMN1 mutations. Zolgensma uses self-complementary adeno-associated virus serotype 9 (scAAV9) to deliver functional SMN1 genes, enabling sustained production of survival motor neuron protein.

SMA is the leading genetic cause of infant mortality under age two, causing progressive muscle atrophy and respiratory failure. In 2019 Zolgensma became the first approved gene therapy for SMA in the US, demonstrating durable efficacy in clinical trials with 92% of treated patients achieving milestone motor functions like sitting independently. The China filing expands access to this potentially curative treatment in a market where over 3,000 new SMA cases are diagnosed annually.

PharmCube's NextBiopharm® database shows that Zolgensma sales have not experienced significant growth since 2021. Click here to request a free trial for NextBiopharm®.