RWD129 Cost Effectiveness of Onasemnogene Abeparvovec in Infants with Presymptomatic Spinal Muscular Atrophy in Italy

Spinal muscular atrophy (SMA) is a devastating rare disease and the most common genetic cause of infant death. Patients with SMA are lacking survival motor neuron 1 (SMN1) gene, leading to reduced SMN protein, loss of functional motor neurons, and progressive, debilitating and often fatal muscle weakness. Though SMN1-targeting treatments like onasemnogene abeparvovec, a one-time gene therapy infusion, can be costly, they provide significant motor improvements in patients with SMA. We sought to assess the cost effectiveness of treatment with onasemnogene abeparvovec versus other disease-modifying agents in infants with genetically confirmed, presymptomatic SMA.

Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: I. Valentini, G. Ghetti, M. Pane, A. Cicchetti, F. Rumi, E. Di Brino, M. Basile, G. Pistillo, M. Bischof Source Type: research