Expanded access for Zolgensma following final NICE guidance

The National Institute for Health and Care Excellence (NICE) has published its final guidance on Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma.

The final guidance recommended the use of Zolgensma (onasemnogene abeparvovec) for use in the NHS as a cost-effective treatment option for infants with SMA Type 1 aged up to 12 months and for babies without symptoms who have a genotype predictive of SMA Type 1.

Under the managed access agreement between Novartis and NICE, Zolgensma is subject to a confidential commercial agreement with NHS England.

In March, NHS England announced that it would provide Zolgensma for SMA Type 1 patinets beyond the expected NICE recommendations but within the marketing authorisation.

According to Novartis, this includes patients who have previously received another treatment and those older than 12 months. A national multidisciplinary team will determine if Zolgensma treatment would be clinically appropriate in such a situation.

Following the final guidance, NHS bodies in Wales and Northern Ireland are also now expected to consider providing access to Zolgensma. The gene therapy is already funded by the NHS in Scotland.

NICE will also evaluate further evidence for the benefits of treating pre-symptomatic babies with Zolgensma as part of the managed access agreement, when this information becomes available – this will include findings from the ongoing SPR1NT clinical trial.

“We are delighted with today’s final guidance by NICE on the use of our one-time gene therapy, Zolgensma, which is a hugely important step forward for families affected by SMA,” said Sally-Anne Tsangarides, general manager at Novartis Gene Therapies in the UK.

“This follows our landmark agreement with NHS England enabling access to this innovative gene therapy on the NHS. We will continue to support clinician education and projects to evaluate newborn screening for SMA to enable the earliest possible diagnosis and support positive outcomes, as well as maintain a dialogue with health authorities and the SMA community to explore ways to expand access for other patients who are included in the marketing authorisation,” she added.