Risdiplam (Evrysdi – Genentech), a survival of motor neuron 2 (SMN2) splicing modifier, has been approved by the FDA for oral treatment of spinal muscular atrophy (SMA) in patients ≥2 months old. It is the first oral drug to be approved in the US for treatment of SMA; nusinersen (Spinraza), an intrathecally administered SMN2-directed antisense oligonucleotide, and onasemnogene abeparvovec (Zolgensma), an IV adeno-associated virus vector-based gene therapy, were approved earlier.

On August 6, 2019, the FDA issued a statement on data manipulation/inaccuracy issues with Zolgensma, a virus vector-based gene therapy approved for one-time IV treatment of children <2 years old with spinal muscular atrophy.1 We reviewed Zolgensma in our July 29 issue.2 According to the FDA statement, the manipulated data pertained to the production process for the product and did not affect the efficacy or safety results from the human clinical trials. It does not require any change in our review.

1. FDA Statement. Statement on data accuracy issues with recently approved gene therapy. Available at: www.fda.gov. Accessed August 15, 2019.
2. Zolgensma – one-time gene therapy for spinal muscular atrophy. Med Lett Drugs Ther 2019; 61:113.

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The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma – Avexis), an adeno-associated virus vector-based gene therapy, for one-time IV treatment of children <2 years old who have spinal muscular atrophy (SMA) and bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. It is the first gene therapy and the second drug to be approved in the US for SMA; nusinersen (Spinraza) was approved in 2016.