Mother seeks £3.05m for son’s rare disease treatment

Tammy McDaid, from Swansea, is seeking access to Fayuvi for her five-year-old son Tate, who has Sanfilippo syndrome type A, a rare inherited condition often described as childhood dementia.

The progressive disease can cause children to lose abilities including speech, movement and eating independently. There is no cure. McDaid hopes treatment could help Tate retain his mobility for longer.

“He doesn’t speak, he’s never spoken. But his mobility is everything to him and that is why I’m fighting for it,” she told the BBC.

A fundraising page set up on her behalf estimates the treatment cost at £2.95 million, with another £100,000 potentially needed for medical expenses, travel, accommodation and follow-up care. It says those estimates still need to be confirmed by healthcare providers. The BBC reported on 8 November that McDaid had raised more than £160,000.

Ultragenyx, the company behind Fayuvi, has set a US list price of $3.95 million for the drug. That is not a confirmed bill for Tate. The company told the BBC it could not give a single estimate for an international patient’s total care costs, which would depend on their medical needs and the treating institution.

The US Food and Drug Administration approved the one-off gene therapy in September for neurological symptoms of Sanfilippo syndrome type A in children with preserved neurodevelopmental function. Reuters reported that, according to the FDA, children treated in trials maintained or improved cognitive function compared with untreated patients. Whether Tate is eligible would depend on assessment by a treating centre.

Ultragenyx said on 6 November that its application for UK authorisation was under review, having been accepted by the medicines regulator on 28 October. Review does not guarantee approval. In October, the MPS Society said there was no formal NHS pathway for the therapy and outlined steps for families considering privately funded treatment in the US.

Staff at Noah’s Ark Children’s Hospital in Cardiff are discussing possible access with US officials on the family’s behalf, the BBC reported. The outcome is not yet clear.