Fourth JCA available: onasemnogene abeparvovec (Itvisma®) for the treatment of 5q spinal muscular atrophy (SMA)

For the active substance onasemnogene abeparvovec (Itvisma®) for the treatment of 5q spinal muscular atrophy (SMA) in patients aged 2 years and older with a biallelic mutation in the survival motor neuron 1 gene (SMN1), the new JCA considered a total of five studies and defined six subpopulations and 14 PICOs.

hta_jca_mp_202412_onasemnogene-abeparvovec_report_en.pdf