Kerry family 'over the moon' as baby Theo in line for multimillion 'miracle drug'

Theo Whelan, who was born last April, is a possible recipient of the 'miracle drug' for the treatment of spinal muscular atrophy

Theo, Shane and Stephanie Whelan at home in Killorglin, Co Kerry.

The family of a child with a rare muscular condition has said they are “over the moon” the HSE has given the approval to reimburse what has been labelled a “miracle drug”.

On Friday, the HSE announced it had approved the gene therapy drug Zolgensma for reimbursement in the treatment of spinal muscular atrophy (SMA) for patients who meet the specified criteria.

SMA is a rare, genetic neuromuscular condition causing progressive muscle wasting and weakness leading to loss of movement, which may affect walking and upper body movement, breathing and swallowing.

The treatment has been hailed as a 'miracle drug' that can lead to vastly improved quality of life and lengthened lifespan.

€2m for one-off treatment

It had previously been rejected by health authorities in Ireland on the basis of cost. It costs about €2m for the one-off treatment and can only be administered to children typically under the age of two, based on their weight.

Theo Whelan, who was born last April in Tralee General Hospital, and lives with his parents, Shane and Stephanie, in Killorglin, Co Kerry, is a possible recipient of the drug.

Theo's father, Shane, who had been campaigning for approval of the drug, said Friday’s announcement comes as a great relief.

“First of all, we have to wait for the antibodies to come back. There’s a test for antibodies, we have that in the next week, which will mean he is eligible for the treatment,” he said.

And once he gets the treatment, it means we can just focus on him, we won’t have to focus on battling for the drug that his condition needs.” 

Theo is currently in hospital because he had an acute respiratory incident.

“He’s recovering really well but we need to get him a bit stronger for this treatment,” Shane said.

The treatment would hugely improve Theo's quality of life, particularly in relation to feeding muscles and his respiratory system, he added.

Shane thanked all of the people online and in person who have helped them fight for access to the drug.

“It really was a community-driven campaign. They understood the big picture of why he needed it," he said.

"They understood it's the best treatment for him and that it needed to be available in Ireland. Thankfully, it is now at the right time.” 

Theo is currently in hospital because he had an acute respiratory incident.

The reimbursement of Zolgensma is the first joint agreement on the price of a drug between Belgium, Ireland and the Netherlands, through the BeNeLuxA Initiative.

The new innovative gene therapy treatment was granted EU marketing authorisation for treating SMA in May 2020.

Price negotiation

The joint process for Zolgensma consisted of a health technology assessment, followed by a price negotiation which commenced in July 2021, the HSE said.

Speaking on Friday, Health Minister Stephen Donnelly said the reimbursement of the drug by the HSE represents a “great achievement” in ensuring access to this gene therapy for two groups of young SMA patients.

“This is a welcome decision for their families, carers and friends,” he added.

Paul Reid, chief executive of the HSE, said the executive as its standard, engages in commercial negotiations with drug manufacturers “to ensure we obtain the best possible value for new medicines”.

“This enables the HSE to reimburse as many medicines as possible, for as many patients as possible, within its resources. HSE decisions on which medicines are reimbursed by the taxpayer must be made on objective, scientific and economic grounds,” he said.

“Providing funding for new high-cost innovative medicines alongside the many demands of the health service presents a continual challenge and the BeNeLuxA engagement with international colleagues aims to collectively address this challenge.”