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BioWorld - Thursday, December 18, 2025
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Home » NICE rejects Orchard’s rare disease drug, but Zolgensma U-turn offers hope
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NICE rejects Orchard’s rare disease drug, but Zolgensma U-turn offers hope

July 9, 2021
By Richard Staines
NICE has said “no” to regular NHS funding for a rare disease gene therapy from Orchard Therapeutics Ltd. in draft guidance – although experience from Novartis AG’s pricey Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy shows this could change. That’s because this week NICE published final guidance that recommends funding for Zolgensma, thought to be the world’s most expensive drug, after an initial rejection late last year.
BioWorld Regulatory Gene therapy Rare diseases Europe NICE

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