Mirum Pharmaceuticals
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The list honors 50 life sciences employers of choice. Employees at SERB Pharmaceuticals and CSL Behring share why their companies stand out.
The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
While falling short of statistical significance, Incyte and Mirum Pharmaceuticals’ ALK2 inhibitor showed a “clear benefit” in reducing abnormal bone formation in a Phase 2 study of fibrodysplasia ossificans progressiva.
BioSpace has named 50 life sciences companies to its 2026 Best Places to Work list. AbbVie, Amneal Pharmaceuticals and BridgeBio executives share what makes their organizations special.
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
Wednesday’s FDA approval expands Mirum’s Livmarli into the rare genetic disorder that causes progressive liver disease. The biotech has also filed a supplemental New Drug Application for a higher dose of the drug and allowing its use in younger patients.
The FDA’s busy week ahead involves three decision dates for potential industry firsts and a highly anticipated advisory committee meeting for two CAR-T therapies.
Mirum Pharmaceuticals announced that Livmarli oral solution met its primary endpoint in the Phase III MARCH study in young patients with progressive familial intrahepatic cholestasis.
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