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Orphans Dominate New US FDA Approvals, Thanks To Rethymic, Tavneos, And Livmarli

Executive Summary

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.

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Premature PRV: US FDA Mistakenly Announces Voucher Award For Enzyvant Ahead Of Approval

Congenital athymia treatment Rethymic (RVT-802) is still under FDA review, with an 8 October user fee goal date, but FDA announced the regenerative medicine product had earned a rare pediatric disease priority review voucher; agency says the notice was published in error and will be withdrawn.

Mirum Targets $500m Sales For Just-Approved Rare Liver Disorder Drug

Regulators have approved Mirum's IBAT inhibitor as the first medication for a genetic disorder which affects up to 2,500 children in the US.

ChemoCentryx’s Avacopan May Need Another Trial After Tepid Advisory Committee Vote

Near tie goes to the US FDA? While almost every member of the Arthritis Advisory Committee agreed with agency’s assessment that ChemoCentryx’s trial of avacopan for ANCA vasculitis was poorly designed, making it hard to tease out the drug’s benefit-risk profile, about half were in favor of approval due to the potential to spare patients from the adverse effects of steroids. Others said the sponsor didn’t meet the agency’s standard for approval off just one pivotal study.

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