Focus of the Report: This report evaluates the efficacy and safety of the gene replacement therapy onasemnogene abeparvovec-xioi (Zolgensma or AVXS-101) for treatment of patients with spinal muscular atrophy (SMA) type 1.

Technology Description: Onasemnogene abeparvovec is a single-dose, intravenous gene replacement therapy for patients with SMA. The goal of onasemnogene abeparvovec therapy is to deliver a functional copy of the human survival motor neuron gene (SMN) to target cells and provide sustained therapeutic benefit through restored expression of survival motor neuron protein. Early restoration of this protein via introduction of a functional copy of SMN may prevent the loss of motor neurons and subsequent muscle degeneration in patients with SMA.

Controversy: Infants diagnosed with SMA type 1 are not expected to live beyond early childhood, and there are few options for life-extending therapy. Published data on the efficacy and safety of onasemnogene abeparvovec are currently quite limited, although if treatment is confirmed to be beneficial, this would represent a major clinical impact for patients and caregivers. A noteworthy point of controversy is that onasemnogene abeparvovec carries a high cost, which may present and barrier to treatment.

Key Questions:

  • Is Zolgensma effective in treating SMA in infants and children?

  • How does Zolgensma compare with other treatments for SMA?

  • Is treatment with Zolgensma safe?

  • Have definitive patient selection criteria been identified for Zolgensma?

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