Funding for onasemnogene abeparvovec, concerned with babies with spinal muscular atrophy
NICE has published the final draft guidance on recommendations for regular NHS funding for onasemnogene abeparvovec.
This therapy is a potentially curative, one-time gene therapy to treat spinal muscular atrophy (SMA), a rare genetic condition. Used specifically for infants up to 12 months of age before symptoms appear.
Also known as Zolgensma, this drug is manufactured by Novartis Gene Therapies. This recommendation comes after NICE reviewed the evidence to determine whether it could be used for regular NHS funding in England and Wales.
Onasemnogene abeparvovec is designed to address the genetic cause of SMA and its effects are believed to be lifelong.
NICE’s initial guidance, released in 2021, recommended it for infants before they developed symptoms of SMA as part of a Managed Access Agreement (MAA). The agreement allows infants to be treated with the onasemnogen abeparvovec while clinical trials are underway to collect more data.
Left untreated, presymptomatic SMA can develop into one of several different types of SMA. Evidence suggests it is particularly effective for presymptomatic SMA in babies. I heard.
SMA is a rare neuromuscular disease caused by genetic mutations that lead to motor neuron dysfunction. It also affects the nerves in the spinal cord that control movement. This results in muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
NICE plans to issue final guidance on onasemnogene abeparvovec for the treatment of presymptomatic SMA next month.
It is estimated that about 40 babies are born each year with asymptomatic SMA.