HSE approves ‘life-changing’ drug for children with spinal disease

The Health Service Executive has approved for use under reimbursement a new “game-changer” gene therapy drug for the treatment of children with a genetic neuromuscular condition called spinal muscular atrophy (SMA).

Zolgensma (onasemnogene abeparvovec), one of the most expensive drugs in the world, will be available for reimbursement from the HSE from today to patients who meet the specified criteria.

The gene replacement therapy has a one-off treatment cost of $2.1 million in the US. The State reached a price agreement as a member of the BeNeLuxA Initiative, a partnership approach to pharmaceutical policy with Belgium, the Netherlands, Luxembourg and Austria. The scheme aims to secure sustainable access to high quality and affordable medicines for participating countries.

Considerable engagement and negotiation with the manufacturer Novartis Gene Therapies has allowed Belgium, the Republic and the Netherlands to jointly reach an agreement on the price of a drug for the first time, according to the Department of Health.

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The HSE’s chief executive, Paul Reid, said decisions on which medicines should be reimbursed by the taxpayer must be made on “objective, scientific and economic grounds”.

“Providing funding for new high-cost innovative medicines [...]presents a continual challenge, and the BeNeLuxA engagement with international colleagues aims to collectively address this,” he said.

The drug was granted conditional marketing authorisation in the European Union in May 2020. It will be used to treat very young patients with SMA, a rare, genetic neuromuscular condition that causes progressive muscle wasting and weakness. In the most severe cases, children cannot walk and have problems breathing and swallowing.

The therapy is administered only once and alters the genetic barriers to normal muscle and nerve development that occur in a child with SMA.

In 2019, the HSE approved another SMA treatment, Spinraza, for reimbursement after a lengthy campaign by patients.

Minister for Health Stephen Donnelly said the reimbursement of Zolgensma by the HSE represents a “great achievement” which will be welcomed by the families and carers of SMA patients.

The Zolgensma drug is approved by the HSE for reimbursement for patients with a bi-allelic mutation in the SMN1 gene or pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.

‘Game-changer’

Welcoming the news were Children’s Health Ireland, St James’s Hospital and Trinity College Dublin, which were involved in delivering the first ever gene therapy to children in Ireland. The collaboration, led by Dr Declan O’Rourke and Prof Martina Hennessey, involved four Irish children and babies with severe SMA over the past 18 months.

Dr O’Rourke described the HSE’s announcement as a “game-changer”.

“For the families who receive a diagnosis of spinal muscular atrophy it is a devastating blow as they know that without treatment their small babies will not survive to become teenagers. Now they will have access to a gene therapy which dramatically improves their quality and the length of their lives,” he said.

Prof Hennessy said she hoped this marked the beginning of an era that would ensure Irish patients had access to the most innovative and life-changing treatments available anywhere in the world.

“Our collaboration has shown that we can deliver these new therapies successfully at St James’s Hospital which means that other cutting-edge treatments for some of the most difficult conditions can be trialled here ensuring Irish patients have equal access to life-saving medicines,” she said.

The director of the patient representative group SMA Ireland, Jonathan O’Grady, said the agreement brought about a “hugely significant day” for Irish babies with the condition.

The announcement means the “two or three children born each year in Ireland with the most severe form of the disease will now have access to this life-changing medicine”, he said.

To achieve maximum effect it is “vital” the drug is administered as early as possible, he said, so the organisation is calling for SMA to be added to the national list of diseases assessed as part of the neonatal screening process.