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

Novartis Calls For Change In How Germany Assesses Orphan Benefit

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...