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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

Executive Summary

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.

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Cross-country drug access coalition BeNeLuxA is reviewing the costly one-off gene therapy for children with metachromatic leukodystrophy.

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