Mirum Targets $500m Sales For Just-Approved Rare Liver Disorder Drug
FDA Green Light For Alagille Syndrome Therapy Livmarli
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.
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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.
Public Company Edition: With the XBI up more than 9% in April, a few follow-on offerings –$23m to support Gamida Cell’s small product launch and $500m to fund Vaxcyte’s large clinical trial program – have made it to market. Also, China’s CytoMed launched the sixth biopharma IPO in the US this year.
Daily notebook from the J.P. Morgan Healthcare Conference: Turbulent weather matches sentiment about the year ahead, with investors looking for reassurance on the IRA, deal-making and growth strategy from big pharma. Updates from J&J, Biogen, BMS, Merck and more.