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The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.
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The darlings of the weight loss and diabetes spaces, GLP-1 receptor agonists have shown promise against Alzheimer’s in recent studies—with Phase III results expected next year from Novo Nordisk.
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.
President-elect Donald Trump and his incoming administration are unlikely to attempt a wholesale restructuring of U.S. healthcare and could promote M&A activity, but controversial picks like Robert F. Kennedy could impact vaccine sales, experts say.
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Forge Biologics (Forge), a member of Ajinomoto Bio-Pharma Services and a leading manufacturer of genetic medicines, announced a clinical update today on five patients with Krabbe disease that have received FBX-101, an AAV gene therapy, after hematopoietic stem cell transplantation (HSCT).
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Read our takes on the biggest stories happening in the industry.
The HHS secretary recently canceled $500 million worth of BARDA contracts around mRNA vaccine research. But the U.S. government has already spent billions on this work, which has saved millions of lives.
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Analysts at BMO Capital Markets said Centessa’s orexin receptor agonist has “best-in-class” potential for narcolepsy, putting the company in a strong position in the $15 billion market.
Eli Lilly says Indianapolis-based Premier Weight Loss is cracking open auto-injector pens containing its blockbuster drug and repackaging them into separate doses.
Researchers in pharma and beyond have historically glommed onto a limited number of disease targets, limiting innovation. AI could change that.
Akero Therapeutics, 89bio, Boston Pharmaceuticals and more are working to bring novel treatment options for metabolic dysfunction-associated steatohepatitis to a market that could reach $16 billion by 2033.
One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the death of a U.S. teenager, a data monitoring committee concluded that they should continue unchanged.
Roche’s reorganization of Spark Therapeutics is coming more into focus, with nearly 300 employees being let go by the end of this year. Spark also trimmed its staff in 2024.
While it’s not unusual for certain positions to turn over with a new administration, the number of senior-level FDA staffers who have recently left the agency is unprecedented. The lack of communication, transparency and human decency is as well.
The FDA has asked for another well-controlled trial to establish the efficacy of reproxalap in dry eye disease.
The FDA derives just under half of its yearly funding from pharma user fees, which help support its operations and fund employee salaries. An analysis from AgencyIQ suggests that the agency is dangerously close to losing it all.
In the Phase III MITIGATE trial, Uplizna cut IgG4-related disease flares by 87% versus placebo.