ONASEMNOGENE ABEPARVOVEC

The PBAC recommended onasemnogene abeparvovec intrathecal (ONA IT) for the treatment of symptomatic spinal muscular atrophy (SMA) in patients at least 2 years of age who have experienced at least two of the defined signs and symptoms of SMA Type I, II or IIIa prior to 3 years of age. The PBAC acknowledged that some patients missed the opportunity to receive ONA IV due to their age and timing of diagnosis. However, the PBAC considered that the introduction of the Newborn Bloodspot Screening program is likely to reduce the number of patients requiring treatment with ONA after 2 years of age in the future. The PBAC noted that the evidence was limited, because SMA is a rare disease. Nevertheless, the PBAC considered that ONA IT is likely to provide similar benefits to the alternative treatments (nusinersen and risdiplam), including motor function outcomes. The PBAC noted that the submission requested a higher price for ONA IT than the price previously accepted for onasemnogene abeparvovec administered intravenously (ONA IV) for use in patients aged no older than 9 months and in patients weighing up to 21 kg. However, the PBAC considered that the higher treatment cost proposed for older patients was not justified and considered that ONA IT would be acceptably cost-effective at the same cost per patient as ONA IV, with the same outcomes-based RSA conditions applied.