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The FDA has introduced models intended to accelerate rare disease drug development, but recent reversals of guidance from the agency speak to a lack of clarity in its implementation. AI can help focus this process.
European pharma companies splashed billions of dollars into the U.S. biopharma sector in a matter of days, but there are differing views on whether the activity represents the rise of a new buyer class or a quirk of timing.
Three pharma CEOs joined the $30 million compensation club in 2025 but Eli Lilly’s David Ricks exceeded his nearest peer by more than $4 million.
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The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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Tuesday, AbbVie partnered with Anima Biotech to discover and develop mRNA modulators in a collaboration of up to $582 million. The partnership will focus on three oncology and immunology targets.
Neurocrine Biosciences and Voyager Therapeutics inked a CNS-targeted collaboration potentially worth a potential $4.4 billion.
Twist Bioscience announced a partnership with Astellas Pharma on Monday, seeking to discover antibodies against several targets to hit on curative therapies for hard-to-treat diseases.
Takeda and Arrowhead Pharmaceuticals’ Phase II trial studying fazirsiran, an investigational RNA therapeutic developed for AATD-LD, was effective in Phase II–but so was the placebo.
Day One Biopharmaceutical reported positive topline data from a Phase II trial in pediatric low-grade glioma, confirming earlier results.
Italian biopharma Chiesi Farmaceutici SpA announced Sunday it was acquiring rare disease-focused Amryt Pharma in a deal that could reach up to $1.48 billion in value.
The pharma titan adds an intriguing blood pressure medication to its line up with a buyout of CinCor.
Ipsen bolsters rare disease portfolio with acquisition of liver disease specialist Albireo.
The FDA approved Eisai and Biogen’s lecanemab (Leqembi) Friday afternoon. It is the second anti-amyloid antibody to be approved for Alzheimer’s disease in two years.