There's finally a treatment for my five-year-old's dementia - but it'll cost me £3m — SkimNews
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- FAYUVI was approved by the FDA in September as the first gene therapy to treat Sanfilippo Syndrome in children, priced at $3.95 million for the one-time treatment — about £2.95 million for Tate's care.
- Tammy McDaid has raised more than £160,000 since beginning fundraising before approval, covering roughly 5% of the estimated treatment cost, and says families outside the US face an unresolved question of who pays.
- Tate McDaid, diagnosed at four and turning six this month, has begun showing signs of cognitive decline; he has never spoken, his mobility is what his mother is fighting to protect, and the condition can progress more rapidly after age five.
- Ultragenyx, which developed FAYUVI, said there is no single estimate for the total cost of care for an international patient because costs depend on individual medical needs and the treating institution, and said it is pursuing a global regulatory strategy.
- Noah's Ark Children's Hospital staff in Cardiff, alongside Welsh health officials, are in discussions with US officials about access pathways for Tate, while the treatment is not yet available in the UK.
- Society for Mucopolysaccharide Diseases estimates about 140 children in the UK have Sanfilippo Syndrome — Tammy's account cites just over 100 — and says there is currently no cure for Type A, though FAYUVI can delay onset and let patients remain active longer.
Why it matters: FDA approval has not translated into access for the roughly 140 UK children with Sanfilippo Syndrome: FAYUVI costs $3.95 million per child, Ultragenyx has quoted no international total, and the McDaids have raised only £160,000. For Tate, who is already showing cognitive decline, cost is the binding constraint on a treatment unavailable in the UK.
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