Mirum Pharmaceuticals, Inc.
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Latest From Mirum Pharmaceuticals, Inc.
Only two breakthrough-designated novel agents remain on the US FDA drugs center’s 2023 user fee calendar, while CBER stacks up on vaccines and gene therapies under both BTD and RMAT programs.
The French company plans to refile Bylvay for Alagille syndrome under a new brand name after the EMA's Committee for Orphan Medicinal Products said the drug did not meet its 'significant benefit' criteria.
Deal Watch: Sangamo Inks Significant Option Deal With Prevail After Losing Tie-Ups With Novartis, Biogen
Plus deals involving Novartis/DTx, Neurogene/Neoleukin, Travere/Mirum, Sanofi/Scribe, Korro Bio/Frequency, MaxCyte/viTToria and more.
Ipsen's oral ileal bile acid transport inhibitor Bylvay is now approved for pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestaisis. However, the biggest opportunity is a third indication – biliary atresia – which could contribute about half of the €800m peak sales predicted by analysts at Jefferies.
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