Novartis Achieves Pivotal Trial Success for Intrathecal Zolgensma in Older SMA Patients After FDA Delay

Background:
Novartis faced a setback in 2020 when the FDA required a pivotal phase 3 trial for the intrathecal formulation of Zolgensma, aimed at treating older spinal muscular atrophy (SMA) patients, instead of accepting a filing based on a phase 1/2 trial14.

Current Status:
Novartis has now reported a positive readout from the pivotal phase 3 trial for intrathecal Zolgensma, which could boost the gene therapy's prospects5.

Market Impact:
The delay initially benefited competitors like Biogen's Spinraza and Roche's Evrysdi, which are approved for broader age groups and SMA subtypes14.

Regulatory History:
Zolgensma, the world's most expensive medicine at $2.1 million, was initially approved by the FDA in 2019 for treating SMA in pediatric patients less than 2 years old24.

Future Prospects:
The success of the pivotal trial could lead to an expanded approval for Zolgensma, potentially reaching patients up to 5 years old and increasing its market share15.

Sources:

1. https://www.fiercepharma.com/marketing/relief-for-biogen-and-roche-novartis-plan-for-zolgensma-older-patients-hits-setback-fda

2. https://www.fda.gov/vaccines-blood-biologics/zolgensma

4. https://www.spglobal.com/marketintelligence/en/news-insights/latest-news-headlines/novartis-new-zolgensma-formulation-faces-delay-as-us-fda-requests-further-study-60454843

5. https://qtxasset.com/cdn-cgi/image/w=384,h=216,f=auto,fit=crop,g=0.5x0.5/https:/qtxasset.com/quartz/qcloud5/media/image/Novartis1.jpg?VersionId=Gt7d1TxlRUMNUgeNbmUMjzzQ210pl3Ly&sa=X&ved=2ahUKEwjk8YuNmdyKAxWFlYkEHXYuIEEQB16BAgGEAI