ICER says Spinraza is overpriced and urges Novartis to show leadership with Zolgensma price

The US Institute for Clinical and Economic Review (ICER) has given its verdict on what constitutes a fair US price for two drugs treating spinal muscular atrophy (SMA), an inherited neurodegenerative disease that means many babies never reach their second birthday.

Spinraza (nusinersen) was approved in 2016 for the treatment of SMA in both children and adults, while Zolgensma (onasemnogene abeparvovec) is a gene therapy that has been studied in infants with Type I SMA, and a US Food and Drug Administration (FDA) decision is expected in the first half of 2019.

"Among the companies bringing gene therapies to market, Novartis has a real opportunity to demonstrate scientific and ethical leadership by setting the launch price of Zolgensma in line with the benefits"The ICER finds that a fair price for Spinraza, based on clinical and cost-effectiveness findings, inclusive of any mark-up for providers, is between $72,000 and $130,000 for the first year of treatment when loading doses are required, and between $36,000 and $65,000 for each successive year.