What does it mean now that Zolgensma is approved?

We’ve been following Zolgensma since before it was submitted to the FDA for approval.

Drug name: Zolgensma® (onasemnogene abeparvovec-xioi)
Manufacturer: AveXis Inc., now a Novartis AG Company
Condition: Spinal muscular atrophy (SMA) patients less than  two years of age

Condition overview

SMA is a genetic disease. It affects motor nerve cells in the spinal cord, affecting the ability to move and breathe. It’s rare. SMA affects approximately one in 11,000 babies, and about one in every 50 Americans is a genetic carrier. SMA can affect any race or gender, but occurs more commonly in males.¹ As many as 10,000 to 25,000 children and adults in the United States currently have SMA.

SMA is divided into 5 phenotypic subtypes (0-IV), based on age of onset and highest motor function milestone achieved. Type 0 affects a baby still in the womb and can be fatal before birth and is usually fatal within the first year of life. Type I is usually diagnosed during an infant’s first six months. It is the most severe and most common.² SMA types II, III and IV, are diagnosed after six months and are less severe.²

Zolgensma overview

SMA is caused by a mutation in a specific gene that controls muscles needed to move and breathe. The new gene therapy manufactured by Novartis called Zolgensma, replaces the mutated gene. This one-time only, intravenuous infusion over one hour, fixes the underlying gene mutation that causes SMA.

Zolgensma is designed to increase the protein in SMN1 and prevent further muscle degeneration caused by SMA through²:

• Delivery of a fully functional human SMN gene into target motor neuron cells
• Production of sufficient levels of SMN protein required to improve motor neuron function
• Rapid onset of effect in addition to sustained SMN protein expression

The wholesale acquisition cost (WAC) for the one-time intravenous infusion of Zolgensma is $2.125 million per treated patient. The Institute for Clinical and Economic Review (ICER) said this price falls within the upper bound of their value-based price benchmark range when certain conditions are met, such as treatment before two years of age and the SMA Type 1 version of SMA. Novartis offers insurers the pay-over-time option of $425,000 a year for five years. Novartis also encourages comparisons of the drug’s one-time cost to that of several years of chronic SMA therapy.

Date approved: May 24, 2019
Benefit: Zolgensma will impact the medical benefit

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References

1. What is SMA? Cure SMA. (n.d.). Retrieved Oct. 16, 2018, from http://www.curesma.org/sma/about-sma/
2. Types of SMA. Retrieved Nov. 2018, from https://www.avexis.com/research-and-development