It's Official: Novartis SMA Gene Therapy Zolgensma Is World's Most Expensive Drug
The total cost of Zolgensma is more than $2m – in line with estimates – or $425,000 annually for five years if payers choose an annuity-like payment plan. Novartis is also negotiating value-based reimbursement agreements for infants treated with the one-time infusion.
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Risdiplam’s efficacy across two clinical trials and oral dosing made it a credible threat before its US FDA approval, but its weight-based pricing falls below Biogen’s Spinraza and Novartis’s Zolgensma.
Biogen said some spinal muscular atrophy patients are currently being treated with Spinraza after receiving the Novartis gene therapy – its main competitor – so it will start a study of the risk/benefit in 2021.
The expected launch of BioMarin's Roctavian for hemophilia A is expected to heighten the profile of expensive gene therapies on payers' radar. Specialty drug costs are expected to continue to grow in 2021.