Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma – pricing the one-time treatment at a record $2.125m.
“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr Emmanuelle Tiongson, a Los Angeles paediatric neurologist who has provided Zolgensma to patients under an expanded access program.
Novartis executives have defended the price, saying a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year.
A review in April by the independent Institute for Clinical and Economic Review (Icer), concluded Novartis’s previous $5m-per-patient value estimate for Zolgensma was excessive.
But on Friday, Icer said that based on Novartis’s additional clinical data, the broad FDA label and its launch price, it believed the drug fell within the upper bound of its range for cost-effectiveness.
Novartis is expecting European and Japanese approval later this year. Zolgensma will compete with Biogen’s Spinraza, the first approved treatment for SMA.
Spinraza, approved in late 2016, requires infusion into the spinal canal every four months. Its list price of $750,000 for the initial year and $375,000 annually thereafter was also deemed excessive by Icer.
Wall Street analysts have forecast Zolgensma sales of $2bn by 2022, according to a Refinitiv survey. Spinraza sales hit $1.7bn last year, and are predicted to rise to $2.2bn in 2022.