“Libmeldy is approved for use in the European Union and U.K., although the U.K.’s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today’s exchange rates), BBC News reported in 2022.”
Libmeldy works on metachromatic leukodystrophy, and six months after treatment 19-month-old Teddi shows no signs of it. It only works before symptoms start, which is why her 3-year-old sister did not qualify. The NHS got it only after Orchard Therapeutics cut the £2.8 million list price. A cure that exists on paper and gets rejected on price is a cure for shareholders first.