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Zolgensma Loses Orphan Benefit in Germany After 'Very High Sales'

Novartis Calls For Change In How Germany Assesses Orphan Benefit

 
• By Francesca Bruce

Zolgensma has exceeded the orphan sales threshold in the six months it has been on the market in Germany and the gene therapy must now undergo a full benefit assessment.

Royalty-free stock illustration ID: 357978764 Stack of money on Germany Map. 3D illustration
Zolgensma sales trigger full benefit assessment in Germany • Source: Shutterstock

German pricing and reimbursement authorities have suspended the abridged orphan benefit assessment ofNovartis’s gene therapy Zolgensma, stating that it must now undergo a full benefit assessment to compare the product against other treatments for spinal muscular atrophy (SMA). Sales of the one-time treatment have exceeded the maximum permitted one-year threshold of €50m ($60.6m) for products considered as having orphan status.

Novartis AG said the move showed that one-time therapies such as Zolgensma (onasemnogene abeparvovec) required a new approach. Zolgensma has been dubbed the world’s most expensive therapy, with a...

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