SMA (spinal muscular atrophy) is characterized by muscle weakness, paralysis, and eventual loss of movement. It is associated with extremely poor outcomes; children with SMA Type 1 do not usually live past 2 years. Zolgensma (onasemnogene abeparvovec) is £1.79 million per dose, making it the highest price of a drug worldwide. Now, NHS England and Novartis have come to a confidential agreement regarding the drug. NICE (the National Institute for Health and Care Excellence) has since released a report on Zolgensma, which is expected to benefit approximately 80 patients per year.
According to Meindert Boysen, deputy chief executive and director of NICE’s Centre for Health Technology Evaluation, “The committee concluded that Zolgensma represents an important development in treating SMA that could not only allow babies to gain important motor milestones such as independent sitting and walking, but for some babies who are diagnosed before they have symptoms, it might come close to being a cure.” He adds, “The collaborative effort of all involved in the evaluation has made it possible to recommend a treatment that, at its list price, is the most expensive drug NICE has ever evaluated, allowing the lives of babies who might otherwise have died before their second birthday to be transformed.” Read more here.
(Source: PMLiVE, 3/8/21)