NHS England and Novartis Gene Therapies have signed a ‘landmark’ agreement that will make Zolgensma available for patients on the NHS.

Zolgensma (onasemnogene abeparvovec) is a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1.

SMA is a rare genetic neuromuscular disease caused by a lack of a functional SMN1 gene. This results in the rapid loss of motor neurones, which in turn affects muscle function – if left untreated, SMA type 1 leads to death or the need for permanent ventilation by the age of two in over 90% of cases.

The deal struck between Novartis and the NHS has secured the therapy at a ‘substantial’ confidential discount – Zolgensma usually has a list price of £1.79m per dose.

The terms of the agreement build on draft recommendations from the National Institute for Health and Care Excellence (NICE) to fast-track access for patients while the final guidance is concluded.

As part of the agreement, some children with SMA type 1 who currently fall outside the draft recommendations will also be eligible to be considered for treatment by a national multidisciplinary team (MDT) of experts.

According to NHS England, approximately 80 patients could potentially benefit from Zolgensma treatment each year.

NHS England is also currently working to identify ‘centres of excellence' to provide the full range services required to administer the treatment safely, with new specialist services set to begin treating patients later this year.

“This deal is a life-changer for youngsters with this cruel disease and for their families,” said Simon Stevens, chief executive of NHS England.

“Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers,” he added.

In clinical trials, Zolgensma demonstrated significant and clinically meaningful benefit in both pre-symptomatic and symptomatic SMA type 1.

This included prolonging event-free survival and the achievement of motor milestones, which were sustained for over five years post-dosing.