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336 stories matching this filter · ← front page

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  • 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 · 1h agoNegative↗ source
  • Opinion: Capricor’s Duchenne muscular dystrophy drug and the countless tiny decisions that can make or break a study
    “Regulatory flexibility can be exercised in rare disease without sacrificing rigor,” writes Mindy Leffler, whose son has Duchenne muscular dystrophy.
    STAT · 1h ago↗ source
  • FDA approves Regeneron’s garetosmab for ultra-rare bone-forming disease
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The FDA has granted approval to Regeneron's antibody garetosmab, which targets activin A, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare disorder that causes…
Endpoints · 15h agoApprovalPositive↗ 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 · 16h agoApprovalPositive↗ 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
  • 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
  • BioMarin to acquire Alesta Therapeutics for $275 million to expand bone disease pipeline
    BioMarin Pharmaceutical announced it will purchase Alesta Therapeutics for an upfront payment of $275 million. Alesta is developing an oral small-molecule drug, ALE1, aimed at treating hypophospha…
    Fierce Biotech · 2d agoDealPositive↗ source
  • Leo Pharma to license Tanabe’s oral drug for rare light-sensitive skin disease, paying up to $435 million
    Leo Pharma announced a licensing agreement with Japan's Tanabe Pharma for an oral treatment targeting two genetic skin disorders that cause pain when exposed to sunlight. Under the deal, Leo Pharm…
    Endpoints · 2d agoDealNeutral↗ source
  • BioMarin signs new deal for early-stage bone disease asset
    BioMarin recently halted development of a rare disease drug that it had acquired in a $270 million transaction. Within a week, the company announced another deal to acquire an early‑stage asset aim…
    Endpoints · 1d agoDealNeutral↗ 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
  • Amylyx's avexitide halves hypoglycemia episodes in phase 3 trial for post‑bariatric patients
    Amylyx Pharmaceuticals reported that its GLP‑1 receptor antagonist avexitide significantly reduced dangerous blood‑sugar drops in a phase 3 study. The double‑blind Lucidity trial enrolled 78 indiv…
    Fierce Biotech · 2d agoPositive readoutPositive↗ source
  • Argenx reports Phase 3 success for Vyvgart Hytrulo in autoimmune myositis, despite subgroup shortfall
    Argenx announced that its subcutaneous antibody Vyvgart Hytrulo achieved its primary endpoint in a Phase 3 trial for autoimmune myositis, a rare group of inflammatory muscle diseases. The trial de…
    Endpoints · 2d agoPositive readoutPositive↗ 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
  • Leo Pharma signs $435 million deal with Tanabe for rare skin disease drug dersimelagon
    Leo Pharma announced a licensing agreement with Mitsubishi Tanabe Pharma for worldwide rights to dersimelagon, an oral melanocortin-1 receptor agonist targeting rare genetic skin disorders. The dr…
    Fierce Biotech · 3d agoDealPositive↗ source
  • Merck KGaA's US research site to cut 20 jobs amid broader R&D downsizing
    Merck KGaA's North American division, EMD Serono, announced it will lay off 20 employees at its Billerica, Massachusetts research site. The cuts are slated to take effect in the second half of Oct…
    Fierce Biotech · 2d agoLayoffsNegative↗ source
  • Saudi FDA launches program to accelerate rare-disease drug development
    The Saudi Food and Drug Authority (SFDA) announced a new initiative called the New Accelerator Program for Drugs in Rare Diseases (NADR). The program is designed to speed up the development, evalu…
    raps · 3d agoRegulatoryNeutral↗ 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 · 5d agoApprovalPositive↗ source
  • Two undisclosed gene-therapy deaths raise doubts about China's fast-track trial pathway
    Two recent deaths in China’s early‑stage gene‑therapy trials have drawn international attention. Both patients suffered severe immune reactions after receiving high‑dose viral vector treatments, an…
    Fierce Biotech · 5d agoRegulatoryNegative↗ source
  • BioCryst to rely on external partnerships after ending internal discovery programs
    BioCryst Pharmaceuticals CEO Charlie Gayer told Fierce that the company remains committed to science and research even as it shuts down its internal discovery efforts. Earlier this year the firm’s…
    Fierce Biotech · 5d agoNeutral↗ 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 · 6d agoRegulatoryNegative↗ source
  • European regulators withdraw Amgen's Tavneos over questionable data
    European regulators announced they are pulling Amgen's rare disease drug Tavneos from the European market after reviewing data they deemed incorrect and misleading. The authorities said the data s…
    Endpoints · 6d agoRegulatoryNegative↗ source
  • Eli Lilly moves to curb black-market sales of retatrutide amid rising illicit demand
    Eli Lilly announced steps to address the growing black‑market trade of its obesity/diabetes drug retatrutide, which has seen increasing illicit use. The company is working with law‑enforcement and…
    STAT · 6d agoRegulatoryNegative↗ source
  • Commure ends referral payment program for its AI clinic tools
    Commure, a health-software firm valued at about $7 billion, announced it is ending the referral-payment arrangements it had with partners who promoted its artificial-intelligence products to medica…
    STAT · 6d agoNeutral↗ source
  • Neurocrine's Prader-Willi drug Vykat XR linked to multiple deaths and serious side effects
    A group of physicians and experts in Prader-Willi syndrome warned clinicians about safety concerns with Vykat XR, a drug approved by the FDA in March 2025 to reduce the intense hunger in patients w…
    STAT · 8d agoRegulatoryNegative↗ source
  • Definium's LSD pill shows significant anxiety reduction in Phase 3 trial
    Definium Therapeutics announced results of a Phase 3 trial testing its LSD‑based pill DT120 in patients with generalized anxiety disorder. The study enrolled 214 participants who received a single…
    STAT · 7d agoPositive readoutPositive↗ source