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Libmeldy: ‘Significant Discount’ For World’s Most Expensive Drug Secures English Funding

US Filing Planned In Late 2022 Or Early 2023

 
• By Neena Brizmohun

Orchard Therapeutics has agreed to lower the price of its £2.8m gene therapy for children with metachromatic leukodystrophy following negotiations with the National Health Service. Meanwhile, reimbursement efforts in the rest of Europe are also gaining speed and a US filing is planned.

Genetic Medicine with dna isolated on white.
Libmeldy is a gene therapy for metachromatic leukodystrophy • Source: Alamy

Libmeldy, the one-off gene therapy for children with a rare, life-limiting neurodegenerative disorder, will be made available on the National Health Service in England after Orchard Therapeutics dropped the price it was asking for what is reportedly the world’s most expensive drug.

The NHS said that Libmeldy (atidarsagene autotemcel) would be offered as treatment for metachromatic leukodystrophy (MLD) after it negotiated “a...

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