The FDA approved Zolgensma — Novartis’ gene replacement therapy drug that can cure pediatric spinal muscular atrophy (SMA) in a single treatment. For susceptible little ones and their families, it’s a huge stride in the fight against a leading cause of infant mortality.
Drug pricing watchdog ICER deems the cost appropriate based on initial clinical success. Given Novartis’s previous $4-5M prediction, ICER hails the “fair” price as a win for patients and the health system.
Despite Novartis’ promise of annual payment plans and rebates, some critics point to the lower costs of competitor Biogen’s Spinraza as proof of overpricing. Novartis says its one-time treatment is more cost-effective and less physically taxing than Spinraza’s long-term spinal injections.
Others accuse Novartis of looking to reward investors — given that Zolgensma was largely developed by its recently acquired AveXis.
Broadly speaking, a wholly business-centric mindset posits that any company’s purpose is to maximize shareholder returns. But when (tiny) lives are at stake, it’s hard to just turn the other check.