Category: Pharma & Biotechnology

  • STAT+: Merck suffers a setback in Europe over its new Keytruda formulation

    STAT+: Merck suffers a setback in Europe over its new Keytruda formulation

    By Ed Silverman

    Oct. 7, 2026

    Pharmalot Columnist, Senior Writer

    Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.

    In a setback for Merck, a Dutch court ruled that the company infringed on a patent pertaining to a subcutaneous formulation of its Keytruda cancer treatment and was ordered to halt manufacturing and marketing of the medication in several European countries, including France, Italy, Sweden, Switzerland, and the Netherlands.

    The ruling came as part of a widening patent infringement dispute with Halozyme Therapeutics over the best-selling cancer treatment. Patients requiring Keytruda will still have access to the existing intravenous version, which is not affected by the patent dispute or court order.

    In a statement, Merck said it “strongly” disagreed with the court’s decision.

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  • STAT+: Forbion raises $2.6 billion, its largest fundraise to date

    STAT+: Forbion raises $2.6 billion, its largest fundraise to date

    Promising Duchenne therapies will test FDA flexibility, Doudna-founded Caribou shutting down, and more biotech news

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    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

    Good morning. Today, STAT released our 2026 Wunderkinds, a group of early-career researchers recognized for doing innovative work in health and medicine. I had the pleasure of speaking with one NIH researcher who has been figuring out how GLP-1 drugs work in the brain. But check out all of the profiles here!

    The need-to-know this morning

    • Cerevance said its experimental pill solengepras reduced Parkinson’s patients’ daily off-time, alongside other symptoms, in a Phase 3 trial. The privately held company, which will now talk to regulators about a potential approval, touts the pill as one of the first potential Parkinson’s drugs that does not act directly on dopamine, sparing patients certain side effects.
    • Henri B. Kagan and Kenso Soai were awarded the 2026 Nobel Prize in chemistry for discovering how to manipulate “mirror image” molecules, paving the way for manufacturing more precise and safer drugs.

    Doudna-founded Caribou is shutting down

    Caribou Biosciences is shutting down after failing to raise enough money to fund a late-stage clinical trial of its off-the-shelf CAR-T therapy for lymphoma.

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    1. National Biotech Reporter

      Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

  • STAT+: Pharmalittle: We’re reading about WHO obesity guidelines for kids, DOJ targeting pharma, and more

    STAT+: Pharmalittle: We’re reading about WHO obesity guidelines for kids, DOJ targeting pharma, and more

    By Ed Silverman

    Oct. 7, 2026

    Pharmalot Columnist, Senior Writer

    Good morning, everyone, and welcome to the middle of the week. Congratulations on making it this far, and remember there are only a few more days until the weekend arrives. So keep plugging away. After all, what are the alternatives? While you ponder the possibilities, we invite you to join us for a needed cuppa stimulation. Our choice today is ginseng peach, a favorite from our pantry. Meanwhile, here is the latest menu of tidbits to help you on your way. We hope you conquer the world and have a wonderful day. And as always, please do stay in touch. We appreciate feedback, tips, and criticism. …

    The World Health Organization released its first guidelines addressing the sharp rise in obesity among children and adolescents, Health Policy Watch says. In 2024, 170 million children and adolescents aged 5–19 years were living with obesity, including 70 million children aged 5 to 9 years and 100 million adolescents aged 10 to 19 years. Obesity prevalence in this age group quadrupled since 1990, rising from 2% to 8%. But the WHO does not recommend pharmacological treatment, bariatric surgery, or weight loss devices for children aged 0 to 9 years. For adolescents aged 10 to 19 years, treatment with approved drugs may be considered only when a supervised multimodal lifestyle program has not achieved desired results.  

    Caribou Biosciences is shutting down after failing to raise the money necessary to fund a late-stage clinical trial of its off-the-shelf CAR-T therapy for lymphoma, STAT notes. The biotech, borne from the research lab of CRISPR scientist and Nobel laureate Jennifer Doudna, will discontinue clinical development of its two CRISPR-based CAR-T therapies, including vispa-cel for the treatment of advanced B-cell non-Hodgkin lymphoma. Over the past year, Caribou had worked successfully with the Food and Drug Administration to finalize the design of a Phase 3 trial for vespa-cel. Unfortunately, raising the money to pay for the study proved too difficult.

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  • STAT+: Justice Department targets drugmakers in new directive on combating white-collar fraud

    STAT+: Justice Department targets drugmakers in new directive on combating white-collar fraud

    By Ed Silverman

    Oct. 7, 2026

    Pharmalot Columnist, Senior Writer

    Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.

    In a warning to life sciences companies, the Department of Justice last week issued a new directive for combating fraud that made a point of noting the agency will pursue violations of a federal law that tightly regulates the pharmaceutical industry.

    Typically, the agency has pursued health fraud cases centered on providers, clinics, home health agencies, and hospices. By expressly including the Federal Food, Drug, and Cosmetic Act in the directive, the National Fraud Enforcement Division appears to be placing drug and device makers, among others, more clearly in its crosshairs.

    “This is a subtle but significant expansion of their jurisdiction,” said Kevin Lowell, a member of the Miller & Chevalier law firm, who spent a decade at the DOJ, where he most recently had been a deputy chief in the National Fraud Enforcement Division’s health care fraud section.

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  • STAT+: In Duchenne muscular dystrophy, a promising therapy is available to a fortunate few

    STAT+: In Duchenne muscular dystrophy, a promising therapy is available to a fortunate few

    By Jason Mast

    Oct. 7, 2026

    General Assignment Reporter

    There’s a boy like you in Minnesota, Yannick’s mom told him a few years ago. He has the same disease. He has the same kind of smile. Maybe you could talk?

    They clicked. After school and on weekends, the two boys boot up their computers, Yannick in San Diego and Brecken in a suburb of St. Paul, Minn. They play Roblox or build virtual worlds on Minecraft: a zoo, for example, like the real-world one 13-year-old Yannick wants to build when he grows up, full of lions, tigers, and giraffes. 

    They seldom talk about Duchenne muscular dystrophy, the rare fatal muscle-wasting disease they live with. But lately it’s all their moms can talk about. In stolen phone calls on the way to coffee or back from school, they compare the two boys: how quickly Yannick is declining and how steadily Brecken is — shockingly — improving.

    Last winter, Brecken, 12, started receiving an experimental drug developed by Avidity Biosciences. The medicine makes the cell’s protein-building machinery “skip” part of the gene, called an exon, behind Duchenne, allowing patients to make a shortened but functional version of a protein essential for muscle survival. In trials, it appeared to virtually arrest the disease in some patients. 

    The approach, however, is out of most patients’ reach. In principle, “skipping” strategies could benefit about 70% of the 10,000 to 15,000 Duchenne boys and men in the U.S. (The disease almost exclusively affects males.) But the medicines have to be targeted to a patient’s specific mutation. Only about 7% of patients — or roughly 900 Americans — are eligible for Avidity’s drug, called Del-zota, which is now under Food and Drug Administration review.

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  • STAT+: AstraZeneca opens new Boston site 

    STAT+: AstraZeneca opens new Boston site 

    Utah pilots AI service to prescribe acne treatment, drug price disclosures are coming, and more biotech news

    HECTOR RETAMAL/AFP via Getty Images

    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Allison covers what hot startups are getting up to, and who’s financing them. She is also co-host of the weekly biotech podcast, “The Readout Loud.” You can reach Allison on Signal at AllisonDeAngelis.01.

    Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

    Good morning. From DC to Boston to Utah, we’ve got news from all over today.

    The need-to-know this morning

    • BridgeBio said the FDA granted priority review to its oral treatment for children with achondroplasia, a common form of dwarfism. An approval decision on the drug, called infigratinib, is expected by Feb. 4, 2027. 
    • Japan’s Shionogi has acquired for $2 billion the U.S.-based biotech IntraBio, the maker of an approved drug that treats the neurological symptoms of two rare diseases. 
    • Arrivent Biopharma said its experimental treatment for exon 20-mutated lung cancer failed to delay tumor progression in a Phase 3 study.

    Trump admin says drug price disclosures are coming

    Trump administration officials said yesterday they will start developing a system for health insurers to disclose prescription drug prices, and a “hopeful” final plan will come out by next May.

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    1. National Biotech Reporter

      Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    2. Biotech Startups and Venture Capital Reporter

      Allison covers what hot startups are getting up to, and who’s financing them. She is also co-host of the weekly biotech podcast, “The Readout Loud.” You can reach Allison on Signal at AllisonDeAngelis.01.

  • STAT+: Are stem cell treatments about to have their day?

    STAT+: Are stem cell treatments about to have their day?

    You’re reading STAT’s D.C. Diagnosis newsletter

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    John writes about the politics of health care, and he is the author of the twice-weekly D.C. Diagnosis newsletter. You can reach John on Signal at John_Wilkerson.07.

    You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays.

    Celebrity doctor Mike Varshavski has been contacted by The Bachelor for its 30th season. Send news tips and hot physicians to [email protected] or John_Wilkerson.07 on Signal.

    A lame lame-duck

    On Friday, President Trump said he will give $90 to more than 20 million seniors to help pay for Medicare premiums.

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    1. John writes about the politics of health care, and he is the author of the twice-weekly D.C. Diagnosis newsletter. You can reach John on Signal at John_Wilkerson.07.

  • STAT+: Pharmalittle: We’re reading about U.S.-funded psychedelic trials, an AstraZeneca R&D center, and more

    STAT+: Pharmalittle: We’re reading about U.S.-funded psychedelic trials, an AstraZeneca R&D center, and more

    By Ed Silverman

    Oct. 6, 2026

    Pharmalot Columnist, Senior Writer

    Rise and shine, everyone, another busy day is on the way. And it is getting off to a pleasant start here on the Pharmalot campus, where clear blue skies and comfy breezes are greeting us. As for the official mascots, they are happily snoozing in their respective corners. As for us, we are firing up the trusty kettle for another cuppa stimulation. Our choice today is honeybush vanilla turmeric. Of course, you are invited to join us. For the full experience, we are now hawking replicas — take a look. Meanwhile, here are a few items of interest. As always, do keep in touch. We appreciate feedback, criticism, and tips. …

    The Trump administration is advancing the first government-funded clinical trials of the psychedelic drug ibogaine to treat opioid addiction and ​post-traumatic stress disorder, Reuters says. The initiative ⁠follows an executive order signed last April by President Trump that dedicated $50 million ​to the federal ibogaine research, as well as recent calls by experts and ​veterans for strict safety standards on psychedelic treatments. The U.S. Food and Drug Administration said it has opened a public docket seeking feedback on trial design, dose selection, patient ​eligibility, safety monitoring, study-stopping rules, and oversight by Nov. 20.

    AstraZeneca’s pivot towards the U.S. market has continued with the opening of a new R&D center in the biotech hotspot of Cambridge, Mass., the centerpiece of a $1 billion investment program in the state, Pharmaphorum explains. The new facility in the Kendall Square area — which is one of the densest biotech clusters in the world, with hundreds of small and large biopharma companies present — covers 570,000 square feet of lab and office space over 18 stories. The center is part of a major investment program by the U.K.-based company that was first announced in 2024 and dramatically expanded to around $50 billion last year.

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  • STAT+: New Trump regulations would clean up health care pricing disclosures

    STAT+: New Trump regulations would clean up health care pricing disclosures

    Public drug price information is now coming next year, Trump officials say

    Mehmet Oz at a federal event on Monday in Washington announcing new health care price transparency rules.Finn Gomez/Getty Images

    By Bob Herman

    Oct. 5, 2026

    Bob Herman is the author of Health Care Inc., an award-winning weekly newsletter about the business of health and medicine.

    Bob covers the money in health care, focusing on health insurance and hospitals. His stories delve into Medicare Advantage, opaque prescription drug benefits, and how much executives actually make. He is also the author of the Health Care Inc. newsletter. You can reach Bob on Signal at bobjherman.09.

    The Trump administration released new regulations Monday that will force health insurance companies to publish cleaned-up spreadsheets of the prices that they pay hospitals and doctors, while stripping out meaningless data that clogged up the files.

    Trump officials also said they will start developing the system for disclosing prescription drug prices next month. A “hopeful” final plan for drug pricing data will come out by next May, said Mehmet Oz, the head of Medicare and Medicaid, at an event Monday. The government will enforce those requirements by the end of 2027. 

    Last year, the Trump administration punted on finalizing drug price disclosure rules, in a victory for drug companies and pharmacy benefit managers that have fiercely guarded their prices. 

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    1. Business of Health Care Reporter

      Bob covers the money in health care, focusing on health insurance and hospitals. His stories delve into Medicare Advantage, opaque prescription drug benefits, and how much executives actually make. He is also the author of the Health Care Inc. newsletter. You can reach Bob on Signal at bobjherman.09.

  • Trump administration plans new probe into global drug pricing

    Trump administration plans new probe into global drug pricing

    • Updated On Oct 22, 2025 at 03:12 PM IST
    • Read by 100 Professionals

    Bengaluru: President Donald Trump’s administration is preparing a new probe into drug pricing practices among U.S. trading partners, a move that would lay the ground for fresh tariffs, the Financial Times reported on Wednesday, citing sources familiar with the matter.

    Reuters could not immediately verify the report.

    The imminent investigation would consider whether U.S. trading partners are underpaying for drugs, the report said, adding that the investigation would come under Section 301 of the Trade Act of 1974.

    The White House did not immediately respond to a Reuters’ request for comment.

    U.S. patients currently pay by far the most for prescription medicines, often nearly three times more than in other developed nations.

    In late September, Trump said the U.S. would impose a 100% tariff on imports of branded or patented pharmaceutical products, unless a pharmaceutical company is building a manufacturing plant in the United States.

    Trump has not followed through on the threat as he presses pharmaceutical companies to lower U.S. drug prices.

    Earlier this year, Trump sent letters to the leaders of 17 major pharmaceutical companies outlining how they should slash U.S. prescription drug prices to match those paid abroad.

    (Reporting by Ananya Palyekar in Bengaluru; Editing by Jacqueline Wong)

    • Published On Oct 22, 2025 at 03:02 PM IST

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