Zolgensma: First Gene Therapy for Pediatric Spinal Muscular Atrophy (SMA)

Zolgensma (onasemnogene abeparvovec-xioi) from AveXis (a Novartis company), is now approved for children less than 2 years of age with spinal muscular atrophy (SMA). SMA is a rare genetic motor neuron disease and most untreated children do not survive past the age of two due to respiratory failure.

Zolgensma is given as a single, one-time infusion and replaces the defective or missing survival motor neuron gene (SMN1) to treat the root cause of SMA and halt disease progression.

In an ongoing clinical trial, effectiveness was demonstrated in 36 patients who were between 2 weeks and 8 months old at study entry. Of 21 patients treated with Zolgensma, there are 19 remaining patients, who range in age from 9.4 to 18.5 months; 13 of these 19 patients are at least 14 months of age. Developmental motor milestones were also met.

Zolgensma can cause acute serious liver injury. In studies, the most common side effects were elevated liver enzymes and vomiting.

Total cost is $2.125 million, according to AveXis, and is considered to be the world's most expensive single-dose, one-time drug.

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