The Zolgensma® (onasemnogene abeparvovec) global Managed Access Program (gMAP) was created in 2020, the first of its kind for a one-time gene therapy, as a pathway while we worked to broaden global access to Zolgensma. The program was available and provided our gene therapy free of charge to eligible patients in countries where it was possible to make Zolgensma available, where it had not yet received approval, or where no access pathway existed.
During this time, nearly 300 children with spinal muscular atrophy (SMA) from 40 different countries were treated free of charge.1 These efforts were part of a broader initiative to address urgent medical needs while working towards sustainable, long-term access solutions in collaboration with local governments and payors.
Now that the global SMA treatment landscape has progressed, we have made the decision after 5 years to close the global program starting with closing for any new patient requests by the end of July 2024 and with the last allocated patient treated by December 2024. gMAP will be closed to all remaining countries. Since the launch of the gMAP in early 2020, the global SMA treatment landscape has progressed. So too, has our work in obtaining sustainable access to Zolgensma – now approved in more than 50 countries and more than 4,000 patients treated globally across clinical trials, managed access programs, and in the commercial setting.
We are continuing working to develop concepts for sustainable partnerships in countries that lack access and that have an interest and willingness to invest in co-creating solutions to broaden access to Zolgensma.
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For general medical information queries, healthcare professionals may submit requests to the Novartis Medical Information team in the US, Europe, Middle East and Asia, Latin America & Canada, or Asia-Pacific.