The mother of a five-year-old boy with a rare form of childhood dementia has to raise nearly £3 million for his life-saving gene therapy treatment, recently approved in the US but not yet available in the UK.
Tate McDaid, from Swansea, was diagnosed with Sanfilippo Syndrome when he was four. The condition, often called childhood dementia, causes gradual loss of abilities such as walking and eating, and about 140 children in the UK have it. In September, the US Food and Drug Administration approved FAYUVI, the first gene therapy for the condition in children. The US price for the one-time treatment was set at $3.95 million, while the treatment was reported to cost £2.95 million per child.
Tammy McDaid said she had spent months praying for the approval. "I was excited when the treatment initially got approved but then reality hit hard the next day," she told BBC Wales. She contacted Ultragenyx, the developer, to understand how families outside the US can afford the treatment. "We understand what they are charging for American citizens, but they've got health insurance. What is it for us in the UK?"
Ultragenyx said it is pursuing a global regulatory strategy and FDA approval is an important first step. It could not provide a single estimate for the total cost of care for an international patient, as costs depend on individual medical needs and the treating institution. The company recommended families work with their child's healthcare provider to explore potential access pathways.
Tammy has been fundraising since before the approval and has raised over £160,000, about 5% of the total estimated cost. “If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away,” she said.
Health officials from Wales were talking to officials in the US about potential access routes for Tate. Staff at Noah's Ark Children's Hospital in Cardiff were discussing this with officials in the US on the family's behalf. Tate turns six this month, and his mother said time is critical. “Tate is already shown signs of cognitive decline,” she added. “The older he gets the less the treatment will work and the more ability he will lose in the process.”
Analysis
Why This Matters
- The £2.95m cost highlights the gap between US drug approval and global access to treatments for rare diseases.
- For UK families like the McDaids, accessing life-saving therapies may require private fundraising or navigating international healthcare systems.
- The case underscores the urgency of international regulatory pathways for gene therapies like FAYUVI.
Background
Sanfilippo Syndrome Type A, also called Mucopolysaccharidosis type III, is a neurodegenerative condition often referred to as childhood dementia. It causes gradual loss of abilities such as walking and eating, and can progress more rapidly after age five. About 140 children in the UK have the condition. There is currently no cure, but the newly approved FAYUVI gene therapy could delay its onset and allow children to remain active for longer. The treatment was approved by the US FDA in September, and its developer Ultragenyx is pursuing global approvals.
Key Perspectives
- Tammy McDaid: says she is fighting against time, has raised over £160,000 but needs millions; the cost is prohibitive and she awaits a response from Ultragenyx on international pricing.
- Ultragenyx: says it is pursuing a global regulatory strategy and FDA approval is a first step; it cannot provide a single cost estimate for international patients and recommends families consult healthcare providers for access pathways.
- Welsh health officials: are discussing potential access pathways with US officials on behalf of the family, with input from Noah's Ark Children's Hospital.
What to Watch
- Developments in talks between Welsh health authorities and US officials that might create an access pathway for Tate.
- Any announcement from Ultragenyx on global pricing or access programs for families outside the US.
- The progress of the family's fundraising campaign and whether it reaches the target before Tate's condition deteriorates further.
Checked
12 claims in this story checked against 1 source: 12 supported.
Show each claim
- SupportedThe headline says Tate’s mother needs £3m for his life-saving treatment for rare childhood dementia.BBC Wales: “The mum of a five-year-old boy with a rare form of dementia has to pay almost £3m for his life-saving treatment.”
- SupportedThe summary says five-year-old Tate was diagnosed with Sanfilippo Syndrome and that his family is raising funds for a gene therapy approved in the US.BBC Wales: “Tate McDaid, from Swansea, was diagnosed with Sanfilippo Syndrome, external - a form of dementia that just over 100 UK children have - when he was four . In September, the first gene therapy to treat the condition in children was approved in the US.”
- SupportedThe treatment had been approved in the US but was not yet available in the UK.BBC Wales: “the treatment that is not available yet in the UK.”
- SupportedTammy said, “I was excited when the treatment initially got approved but then reality hit hard the next day,” to BBC Wales.BBC Wales: “"I was excited when the treatment initially got approved but then reality hit hard the next day," Tammy said.”
- SupportedTammy said, “We understand what they are charging for American citizens, but they've got health insurance. What is it for us in the UK?”BBC Wales: “"We understand what they are charging for American citizens, but they've got health insurance," she added. "What is it for us in the UK?”
- SupportedTammy said, “If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away.”BBC Wales: “She said: "If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away."”
- SupportedTammy said, “Tate is already shown signs of cognitive decline.”BBC Wales: “"Tate is already shown signs of cognitive decline," she added.”
- SupportedTammy said, “The older he gets the less the treatment will work and the more ability he will lose in the process.”BBC Wales: “"The older he gets the less the treatment will work and the more ability he will lose in the process.”
- SupportedIn September, the US FDA approved FAYUVI, the first gene therapy for the condition in children.BBC Wales: “In September, the first gene therapy to treat the condition in children was approved in the US.”
- SupportedThe one-time treatment’s US price was set at $3.95 million, and it was reported to cost £2.95 million per child.BBC Wales: “This was when she read the US price for the one-time treatment, external had been set at $3.95m.”
- SupportedTammy had raised over £160,000, about 5% of the total estimated cost.BBC Wales: “Tammy began raising money before the new drug was approved by the US Food and Drug Administration, and now has more than £160,000. But this covers only about 5% of the total estimated cost of the treatment.”
- SupportedUltragenyx said it could not provide one estimate for an international patient’s total care costs because those depend on individual medical needs and the treating institution.BBC Wales: “There is not a single estimate we can provide for the total cost of care for an international patient, as those costs would depend on the patient's individual medical needs and the treating institution.”
