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Vertex Pharmaceuticals

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The rare disease pricing model is starting to fray
Biotechnology
Rare disease drug pricing has long rested on a simple trade-off. Small patient populations and high development costs justified higher prices, and payers largely accepted that logic because the overall budget impact remained limited. That balance is becoming harder to maintain as orphan drugs now account for a growing share of new launches and an increasing portion of drug spending.   8 April 2026


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Biotechnology
US Biotech company Regenxbio and the University of Pennsylvania on Friday convinced a US appeals court to revive their patent lawsuit against Sarepta Therapeutics over Sarepta's Duchenne muscular dystrophy (DMD) treatment, according to a Reuters report.   23 February 2026