Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug Administration (FDA) announced today, issuing an historic decision.

With this news, Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) became the first gene therapy for SMA and its second disease-modifying treatment after Spinraza(nusinersen, by Biogen), whose approval came just days before Christmas in 2016.

It is seen as potentially “curative” and a “one-time” therapy for infants treated early enough in this disease’s harsh course, company executives and scientists said in repeat interviews.

“Zolgensma’s one-time dose of gene therapy has the potential to make a truly transformative impact on this life-threatening disease,” Kenneth Hobby, president of Cure SMA, a patient advocacy group, said in a press release. “We are excited the FDA’s approval of Zolgensma brings patients and families a powerful new treatment which corrects the underlying cause of the disease.”

The FDA decision was based on data from the ongoing Phase 3 STR1VE trial (NCT03306277), and the completed Phase 1 START trial  (NCT02122952) evaluating the efficacy and safety of Zolgensma, given as an intravenous infusion, to type 1 infants up to 6 months old.

By approving Zolgensma to treat pre-symptomatic newborns through toddlers, the FDA went a bit beyond the approval request filed in October 2018 for type 1 babies up to age 9 months.

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