SMA drug Zolgensma’s label more surprising than its price

FDA’s surprise label for SMA gene therapy Zolgensma gives Novartis cover for its unprecedented price.

FDA surprised Novartis, patient advocates and physicians by approving a label for the pharma’s gene therapy that creates hope that newborns diagnosed with spinal muscular atrophy can be treated before symptoms appear -- in time to head off permanent, severe disability. The label also gives the company cover for a price that would be hard to justify for the narrower population that it studied in the trials to support its marketing application.

On May 24, FDA approved the gene therapy manufactured by AveXis, a unit of Novartis AG, for all patients with SMA under two years old with bi-allelic mutations in the SMN1 gene.

This is a much broader population than was included in the studies that supported approval. A completed trial and an ongoing trial enrolled only symptomatic Type 1 SMA patients with mean ages of 3.4 months and 3.9 months, respectively.

Although it’s not unheard of for FDA to grant a label beyond the study population, this one took the company by surprise.

FDA’s label sets up Zolgensma onasemnogene abeparvovec-xioi to compete with Spinraza nusinersen from Biogen Inc. to treat pre-symptomatic babies diagnosed with SMA. The two

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