Funding is for onasemnogene abeparvovec and concerns babies with spinal muscular atrophy

NICE has published final draft guidance regarding the recommendation of routine NHS funding for onasemnogene abeparvovec.

The treatment is a potentially curative one-off gene therapy for treating a rare genetic condition, spinal muscular atrophy (SMA). It will be used specifically among babies aged up to 12 months, before they develop symptoms.

The drug – also known as Zolgensma – is produced by Novartis Gene Therapies. The recommendation has emerged after NICE reviewed the evidence to determine whether it should be available for routine NHS funding in England and Wales.

Onasemnogene abeparvovec is designed to address the genetic cause of SMA and its effects are thought to be life-long.

Under NICE’s original guidance, released in 2021, it was recommended for babies before they develop symptoms of SMA as part of a managed access agreement (MAA). The agreement ensured that babies could have treatment with onasemnogene abeparvovec while a clinical trial to collect more data was undertaken.

If left untreated, pre-symptomatic SMA develops into one of several different types of SMA. Evidence suggests that it is particularly effective for pre-symptomatic SMA in babies. Meanwhile, the committee heard that it provides substantial health benefits and may in some cases come close to being a cure.

SMA is a rare neuromuscular condition caused by a genetic mutation which leads to motor neurones malfunctioning. It also affects the nerves in the spinal cord which control movement. This causes muscle weakness, progressive loss of movement and difficulty breathing and swallowing.

NICE expects to issue final guidance on onasemnogene abeparvovec for treating pre-symptomatic SMA next month.

It is estimated that about 40 babies are born each year with pre-symptomatic SMA.