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Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
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By far, the largest acquisition of 2024 was Novo Holdings’ yet-to-be-closed buyout of manufacturer Catalent at $16.5 billion. Outside of that, the leading pharmaceutical companies kept to less than $5 billion per deal.
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.
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Takeda and Protagonist Therapeutics, Inc., announced the signing of a worldwide license and collaboration agreement for the development and commercialization of rusfertide, an investigational injectable hepcidin mimetic peptide of the natural hormone hepcidin, currently in a pivotal Phase 3 trial, VERIFY, for the treatment of Polycythemia Vera.
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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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Stifel analysts said that Lexeo’s data showing reduced size and thickness of the heart’s left ventricle are “supportive of a drug effect” for the company’s gene therapy in Friedreich’s ataxia cardiomyopathy.
Senators Bill Cassidy and Bernie Sanders asked HHS Secretary Robert F. Kennedy, Jr. to make good on a pledge to appear before the committee regularly. It is not clear if or when the hearing, which was scheduled for Thursday, will take place.
After moving the manufacturing of a CAR T drug that was part of its ImmPACT Bio acquisition to Washington, Lyell Immunopharma is closing its Los Angeles manufacturing facility, eliminating dozens of jobs there.
Stifel analysts were bullish on the data, which showed a 16.5% drop in body-mass index among patients with damage to the hypothalamus taking Rhythm Pharmaceuticals’ Imcivree.
GSK is paying to access ABL Bio’s Grabody-B platform, which potentially enables therapies to cross the blood-brain barrier.
Under Friday’s final ruling anti-obesity medications for weight-loss will remain ineligible for Medicare coverage.
Kennedy, a long-time opponent of vaccines, stated that the MMR vaccine is “the most effective way” to combat the measles outbreak, which has already claimed the lives of two children in the U.S.
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.