Zolgensma: a remarkable new treatment, an ICER analysis, and a poorly justified price

Health Affairs Blog

18 June 2019 - Defects in both sets of alleles in the coding region for the survival motor neuron 1 protein lead to the rare heritable condition spinal muscular atrophy, a disorder characterised by deterioration of motor neuron cells. 

The clinical presentation is heterogenous, ranging from type 1 disease that causes neuromuscular decline and death, typically before age 2, to type 4, an adult onset condition associated with weakness and a normal lifespan. Fewer than 1,000 infants in the U.S. are born each year with spinal muscular atrophy (SMA).

In May 2019, Novartis obtained U.S. FDA approval for the gene therapy onasemnogene abeparvovec-xioi (Zolgensma). Zolgensma transfects additional copies of the survival motor neuron (SMN) gene into motor neuron cells using an adenovirus vector. In two remarkable single arm clinical trials, infants who had already developed symptomatic type 1 SMA and then received Zolgensma not only survived well past their predicted date of death, but experienced improvement in neuromuscular function. Zolgensma is approved for infants under the age of 2 with any type of SMA, whether symptomatic or not.

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Michael Wonder

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Michael Wonder