The FDA just approved Alyftrek, a once-daily medicine for a small slice of cystic fibrosis patients that carry certain mutations, including F508del. It’s a triple combination CFTR modulator that works across 31 other mutations, and outperformed Trikafta — another popular Vertex drug for cystic fibrosis — in its ability to reduce sweat chloride levels. This is the...
Category: <span>News</span>
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A drug price dynamic to watch
from AXIOS: Reproduced from Peterson-KFF Health System Tracker; Chart: Axios VisualsEven the Medicare-negotiated rates that the U.S. pays for some drugs is generally much higher than what other comparable countries pay for the same products, a new KFF analysis found.Why it matters: Trump seemingly dropped his proposal to drive better bargains for what the U.S. pays for drugs when he...
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Another PD-1/VEGF program sees hefty funding
From my colleague Drew Joseph: Just a few months after its unveiling, the startup Ottimo Pharma said Thursday it had raised a $140 million Series A round, a hefty sum that reflects the biopharma’s industry excitement around the targets Ottimo’s going after with its experimental cancer therapy. The financing was led by OrbiMed, Avoro Capital, and Samsara BioCapital,...
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PBMs took secret payments to allow free flow of opioids
By The New York Times | December 17, 2024 Topics addiction business strategy culture epidemic medical ethics opioids pharmacy regulation For years, P.B.M.s took payments from opioid manufacturers, including Purdue Pharma, in return for not restricting the flow of pills. As tens of thousands of Americans overdosed and died from prescription painkillers, the middlemen collected billions of...
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Woman receives kidney transplant from genetically modified pig
Woman receives kidney transplant from genetically modified pigJoe Carrotta for NYU Langone HealthOver seven hours on November 25, surgeons in New York moved a kidney from a genetically modified pig into a 53-year-old woman who had needed a transplant since 2017. This is the third time doctors have performed a kidney transplant with an organ...
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Scientists urge ban on creating “mirror life”
By Tina Reed Illustration: Sarah Grillo/Axios Scientists are sounding new alarms about man-made organisms whose constituent molecules are mirror images of what’s found in nature and could pose unprecedented risks to humans, animals and the environment.Why it matters: While the ability to create “mirror life” is probably at least a decade away, researchers already have synthesized...
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GLP-1 drugs spark disagreements in Trump’s circle
From STAT’s Elaine Chen: GLP-1 drugs seem to be a source of contention among the people in President-elect Trump’s circle. Earlier this week, Elon Musk tweeted: “Nothing would do more to improve the health, lifespan and quality of life for Americans than making GLP inhibitors super low cost to the public.” It’s a sentiment he’s expressed several times before. (The...
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Self-collection for HPV instead of pelvic exams
Illustration: Aïda Amer/Axios Women at age 30 can start collecting their own vaginal sample for cervical cancer screening at a physician’s office and forego pelvic exams, a federal health advisory committee said on Tuesday. Why it matters: The draft guidance from the U.S. Preventive Services Task Force marks the first time self-collection for human papillomavirus has been...
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1 big thing: Supplement makers seize on GLP-1 boom
By Maya Goldman Illustration: Shoshana Gordon/Axios Some supplement companies are vying to get into the booming GLP-1 space, adding a new layer of complexity to accessing anti-obesity treatments.Why it matters: GLP-1s are expensive and in short supply. Supplements promoted with similar claims may be easier to come by, but experts warn they don’t have to meet...
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Teenage girl gets long-awaited Duchenne treatment
Sarah Jenssen is the rarest of the rare — a girl with Duchenne muscular dystrophy, which almost always afflicts boys, as STAT wrote about in 2019. She wasn’t eligible to enroll in clinical trials for cutting-edge Duchenne therapies, but her family was elated when the FDA this year approved a gene therapy called Elevidys from Sarepta...