Five-month-old Arthur Morgan, who was diagnosed with SMA in May, has received the one-off gene therapy at Evelina London Children’s Hospital.

SMA is a rare genetic disease that causes paralysis, muscle weakness and progressive loss of movement. Children born with SMA type 1 typically have a life expectancy of around two years.

The list price for Zolgensma (onasemnogene abeparvovec) is £1.79m per dose, and has previously been labelled as the ‘most expensive drug in the world’.

The first administration of Zolgensma comes only a few months after Novartis and NHS England reached a landmark deal for the gene therapy.

NHS England said that the gene therapy will be available to patients at a ‘price that is fair to taxpayers’, adding that the deal secured the treatment at a substantial discount, although this has been kept confidential.

Following the deal, the National Institute for Health and Care Excellence (NICE) published a draft recommendation for the gene therapy.

This recommendation advises the use of Zolgensma on the NHS for babies aged up to 12 months with SMA type 1 before they develop symptoms.

NICE has agreed a managed access agreement for Zolgensma, meaning that further data is set to be collected prior to the publication of the final guidance. According to NHS England, up to 80 patients will be able to benefit from treatment with Zolgensma each year.

The gene therapy will also be offered to young children with SMA type 1 who do not meet the recommendation criteria. The use of the potential gene therapy treatment for these patients will be discussed by a national multidisciplinary team.

“It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS. The NHS Long Term Plan committed to securing cutting-edge treatments for patients at a price that is fair to taxpayers,” said Simon Stevens, chief executive of the NHS.

“Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families,” he added.

The four specialist NHS sites that have been commissioned to administer Zolgensma across England are: Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, University Hospitals Bristol and Weston NHS Foundation Trust and Evelina London Children’s Hospital.

A US Food and Drug Administration (FDA) panel has voted in favour of Provention Bio’s type 1 diabetes prevention drug teplizumab, setting it up for a potential approval in July.

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 10-7 in favour of the benefits of teplizumab outweighing its risks to support approval of the drug to delay type 1 diabetes.

The EMDAC based its recommendation on data from the phase 2 TN-10 study, which randomly assigned 76 participants aged eight to 45 to receive a 14-day treatment course of teplizumab or placebo.

In this study, teplizumab demonstrated a significant delay in the onset and diagnosis of the chronic condition. Compared to placebo, children and adults who were considered high-risk of developing type 1 diabetes saw it delayed by a median of two years.

However, some members of the EMDAC questioned the size of the study, noting that the drug was actually tested in only 44 participants.

In addition, Provention Bio also submitted safety data for teplizumab in another indication, with the panelists commenting this could not be used to make a good comparison.

Some EMDAC members also raised concerns regarding the higher rate of serious adverse events among teplizumab-treated patients, compared to placebo – 16% and 3%, respectively.

Provention Bio’s CEO Ashleigh Palmer said: “We know the [type 1 diabetes] community and at-risk patients and their families in particular are waiting urgently for access to clinical advancements to address their significant medical needs. We remain committed to working closely with the FDA to hopefully secure approval of teplizumab and potentially bring the first disease-modifying therapy in T1D to at-risk patients as soon as possible.”

The prescription drug user fee act (PDUFA) action date for a final FDA decision on the biologics licence application (BLA) for teplizumab is due on 2 July 2021.

However, the company added that previously disclosed issues with the pharmacokinetic comparability for the drug – which were not discussed during the EMDAC meeting – are ‘likely’ to result in a delay to the potential BLA approval timelines.