UK’s NICE Authorizes Novartis’ Gene Therapy for Spinal Muscular Atrophy

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Zolgensma (onasemnogene abeparvovec) for babies up to 12 months with type 1 spinal muscular atrophy, a rare genetic disorder with a life expectancy of less than two years.

The gene therapy is the most expensive treatment NICE has ever recommended for the National Health Service use, potentially costing $2.5 million (£1.79 million) per treatment.

NICE said the therapy should be administered to infants with spinal muscular atrophy “before they develop symptoms, as part of a managed access arrangement while further data is collected.”