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324 stories matching this filter · ← front page

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  • Layoffs hit over 1,300 workers across seven biopharma M&A deals in 2026
    Workforce reductions this year have affected at least seven biopharma companies that recently completed mergers or acquisitions, eliminating a minimum of 1,359 jobs, according to BioSpace data. Th…
    BioSpace · 6h agoLayoffsNegative↗ source
  • Capricor Therapeutics likely faces FDA rejection of its Duchenne cell therapy
    Capricor Therapeutics is working to avoid an imminent FDA rejection of its cell‑based treatment for Duchenne muscular dystrophy. The company says it remains confident in the drug, but the regulator…
    STAT · 2h agoRegulatoryNegative↗ source
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  • Capricor’s Duchenne drug deramiocel receives negative FDA advisory vote on heart outcome
    Mindy Leffler, a parent of a young man with Duchenne muscular dystrophy, writes an opinion piece about Capricor’s phase 3 study of its drug deramiocel. She describes her son’s experience living ind…
    STAT · 4h agoRegulatoryNegative↗ source
  • Pediatric rare cancer research draws steady investment despite biotech market volatility
    The article notes that pediatric rare cancers continue to attract investment because they address the greatest unmet medical needs and allow clear measurement of patient impact. Even as the biotec…
    BioSpace · 9h agoPositive↗ source
  • Experts warn protein restriction could harm older adults and GLP-1 patients
    Researchers have recently suggested that limiting dietary protein might improve metabolic health and extend lifespan, a claim highlighted in a review paper in Cell Press Blue. Nutrition specialist…
    STAT · 4h agoNegative↗ source
  • Regeneron's Pasatru receives FDA approval for ultra-rare bone-growth disease FOP
    Regeneron Pharmaceuticals has secured FDA approval for Pasatru, a therapy aimed at treating fibrodysplasia ossificans progressiva (FOP), an ultra‑rare condition in which bone forms in soft tissues.…
    STAT · 20h agoApprovalPositive↗ source
  • FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type Ia
    The U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen…
    fda press · 15h agoApprovalPositive↗ source
  • Takeda’s FDA-approved Orzeyful expands treatment to all narcolepsy symptoms, challenging existing sleep drugs
    The U.S. Food and Drug Administration approved Takeda's drug Orzeyful in August, marking the first therapy that addresses the full spectrum of narcolepsy symptoms, including cataplexy and excessive…
    BioSpace · 1d agoApprovalPositive↗ source
  • Trump nominates White House aide Heidi Overton as FDA commissioner
    President Donald Trump is reported to be selecting Deputy Director of the White House Domestic Policy Council Heidi Overton, a physician, to be the next FDA commissioner, pending Senate confirmatio…
    BioSpace · 1d agoRegulatoryNeutral↗ source
  • Experts split over Huidagene’s gene-editing approach after boy’s death in Duchenne trial
    A young boy died while participating in Huidagene’s gene‑editing trial for Duchenne muscular dystrophy, raising serious questions about the company’s experimental strategy. STAT+ reviewed video of…
    STAT · 1d agoNegative↗ source
  • CLINUVEL to cut up to 20% of staff and move headquarters to New York
    CLINUVEL announced a strategic reorganization aimed at focusing resources on the U.S. market. The company will reduce its global workforce by 10% to 20%, affecting up to 21 of its 104 employees.…
    BioSpace · 23h agoLayoffsNegative↗ source
  • Eyepoint’s Phase 3 trial for age‑related blindness treatment fails primary endpoint, jeopardizing approval plans
    Eyepoint announced that its Phase 3 trial of the experimental drug Duravyu for a common cause of age‑related blindness did not meet its primary endpoint. Patients receiving the investigational ther…
    STAT · 1d agoNegative readoutNegative↗ source
  • LEO Pharma spends $435 million to acquire rare-disease skin drug dersimelagon
    LEO Pharma announced it will pay $435 million to buy the rights to dersimelagon, an oral MC1R agonist originally developed by Tanabe Pharma. The drug has completed Phase 3 trials for two rare phot…
    BioSpace · 1d agoDealPositive↗ source
  • Amylyx to seek FDA approval for avexitide after Phase 3 success in post-bariatric hypoglycemia
    Amylyx announced plans to file a new drug application for avexitide, a GLP-1 receptor antagonist, after a Phase 3 trial showed reduction in serious hypoglycemia in patients who had gastric bypass s…
    BioSpace · 2d agoRegulatoryPositive↗ source
  • AI and data sharing essential for safer cell and gene therapies, says industry leader
    The author argues that keeping data secret in cell and gene therapy harms patients, and that a commitment to transparency and artificial intelligence is needed to make these treatments safer and mo…
    BioSpace · 2d agoPositive↗ source
  • Opinion warns US surrogacy system is chaotic and may face bans after decades of legal battles
    Arthur Caplan writes that the United States surrogacy framework is poorly managed, using the recent West case as a warning sign. He argues that the lack of clear rules could lead to broader bans.…
    STAT · 2d agoNegative↗ source
  • AstraZeneca halts late-stage lung cancer trial of volrustomig after disappointing interim results
    AstraZeneca announced it is stopping a Phase 3 study of its bispecific antibody volrustomig in metastatic non-small cell lung cancer after an interim analysis showed the drug is unlikely to meet it…
    BioSpace · 3d agoNegative readoutNegative↗ source
  • AstraZeneca halts late-stage lung cancer trial of volrustomig after disappointing interim results
    AstraZeneca said it is ending its Phase 3 eVOLVE-Lung02 trial that evaluated the bispecific antibody volrustomig combined with chemotherapy as a first-line option for metastatic non-small cell lung…
    BioSpace · 3d agoNegative readoutNegative↗ source
  • IRS launches probe into UnitedHealth over alleged tax avoidance from 2017-2020
    UnitedHealth Group is under investigation by the Internal Revenue Service, which says the insurer underpaid taxes by moving money to a foreign subsidiary during 2017-2020. The IRS is seeking to in…
    STAT · 3d agoRegulatoryNegative↗ source
  • FDA clears Bristol Myers' Zenbexus, first drug of a new class for advanced multiple myeloma
    The FDA announced approval of an oral therapy from Bristol Myers Squibb for patients with advanced multiple myeloma. The drug, iberdomide, will be marketed as Zenbexus and is intended to be used t…
    STAT · 6d agoApprovalPositive↗ source
  • EU regulator withdraws Amgen's Tavneos authorization over serious trial breaches
    European regulators have withdrawn the marketing authorization for Amgen's rare disease drug Tavneos, citing serious breaches in the pivotal clinical trial. The EMA said study staff from ChemoCent…
    BioSpace · 6d agoRegulatoryNegative↗ source
  • Capricor Therapeutics sees stock surge as FDA signals openness to extend review of DMD cell therapy
    Capricor Therapeutics' CEO Linda Marbán told investors that the FDA is receptive to adding open label extension data to its Biologics License Application for deramiocel, its Duchenne muscular dystr…
    BioSpace · 5d agoRegulatoryPositive↗ source
  • Neurocrine’s rare-disease drug approved but later linked to deaths and serious side effects
    Neurocrine Biosciences received regulatory approval for a rare‑disease therapy after a small clinical trial showed modest efficacy and side effects that were considered manageable. The decision was…
    STAT · 7d agoRegulatoryNegative↗ source
  • Bankrupt Sangamo sells Fabry gene therapy to PTC and prion program to Lilly for $161M
    Sangamo Therapeutics, currently in Chapter 11 bankruptcy, has agreed to divest its Fabry disease gene therapy and several other platforms to raise cash for the restructuring. PTC Therapeutics emer…
    BioSpace · 7d agoDealNeutral↗ source
  • Zealand Pharma sells rights to rare blood cancer drug rusfertide for up to $100M
    Zealand Pharma entered a royalty purchase and sale agreement with Royalty Pharma, receiving $50 million upfront and the possibility of an additional $50 million after one year. In exchange, Zealan…
    BioSpace · 7d agoDealNeutral↗ source