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48 stories matching this filter · ← front page

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  • Regeneron’s ultra-rare bone disorder drug receives U.S. approval after 30-year development
    Regeneron Pharmaceuticals announced that its therapy for an ultra-rare condition in which bone forms in soft tissue has been approved by the U.S. Food and Drug Administration. The disease, sometim…
    STAT · 2h agoApprovalPositive↗ source
  • FDA approves Ultragenyx gene therapy and Regeneron ultra-rare disease drug
    The Food and Drug Administration gave the green light to a gene‑therapy product from Ultragenyx, marking a new treatment option in the company’s pipeline. In a separate decision, Regeneron secured…
    STAT · 1h agoApprovalPositive↗ source
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fda recalls47
fda medwatch36
fda press28
888
Psychiatry & CNS712
Ophthalmology420
Endocrinology & Metabolism378
Pain372
Infectious Disease346
  • 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 · 6h agoRegulatoryNegative↗ source
  • FDA approves Regeneron’s garetosmab for ultra-rare bone-forming disease
    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 · 20h agoApprovalPositive↗ source
  • Merck and Moderna report success in late-stage trial of personalized mRNA cancer vaccine
    Merck and Moderna announced that their personalized mRNA cancer vaccine achieved its primary endpoint in a randomized late-stage trial, marking the first demonstration that such an approach can be…
    STAT · 22h agoPositive readoutPositive↗ 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 · 22h agoApprovalPositive↗ 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 · 2d agoDealNeutral↗ source
  • Safety concerns emerge as radiopharmaceuticals advance in cancer trials
    Radiopharmaceuticals are being hailed for their ability to deliver radiation directly to tumours, potentially sparing surrounding healthy tissue and improving cancer therapy. However, as more cand…
    STAT · 10d agoNegative↗ source
  • FDA grants accelerated approval to Tudriqev viral therapy for PD-1-refractory advanced melanoma
    The U.S. Food and Drug Administration has granted accelerated approval to Tudriqev (vusolimogene oderparepvec-wtpg), a genetically engineered oncolytic viral therapy for patients with advanced, tre…
    fda press · 13d agoApprovalPositive↗ source
  • Call to unify MD and DO medical education pathways
    Physicians Abigail MacKenzie and Vijay Rajput argue that keeping medical education split between allopathic (MD) and osteopathic (DO) tracks is an outdated practice that adds no value to patient ca…
    STAT · 15d agoPositive↗ source
  • Hinge Health to acquire Cylinder Health for $105 million, expanding into digestive care
    Hinge Health, known for virtual physical therapy, announced it will purchase digestive health startup Cylinder Health. The transaction is a cash deal valued at $105 million, marking Hinge's latest…
    Endpoints · 15d agoDealPositive↗ source
  • California Supreme Court rejects claim that Gilead must fast-track safer HIV drug
    Thousands of patients sued Gilead Sciences, alleging the company was negligent for delaying a safer HIV medicine and that they suffered kidney injury and bone loss from the older drug. The Califor…
    STAT · 16d agoRegulatoryNeutral↗ source
  • Ebola cases approach key milestone as vaccine candidates draw near completion
    The latest update on the Ebola outbreak indicates that the number of cases is approaching a significant threshold, while vaccine development efforts are reported to be close to delivering a usable…
    STAT · 21d agoNeutral↗ source
  • A decade after WHO recognition, mycetoma is still one of the world’s most neglected diseases
    Borna Nyaoke-Anoke, a physician leading mycetoma work at the Drugs for Neglected Diseases initiative, reflects on a decade since the World Health Organization formally acknowledged the disease. Sh…
    STAT · 21d agoNegative↗ source
  • FDA selects Dexcom as first participant in TEMPO digital health pilot
    The U.S. Food and Drug Administration announced that Dexcom, Inc. has been chosen as the inaugural manufacturer for the Technology-Enabled Meaningful Patient Outcomes (TEMPO) pilot focused on digit…
    fda press · 28d agoRegulatoryPositive↗ source
  • Study finds rising job dissatisfaction and health‑care access uncertainty among U.S. workers
    A new study shows increasing levels of job dissatisfaction and uncertainty about health‑care access among U.S. workers. The research, funded by AHRQ, surveyed a national sample and found that a gr…
    STAT · 29d agoNegative↗ source
  • Urologist warns that Defense Dept's testosterone screening for servicemembers fuels outdated masculinity myths
    Dr. Elizabeth Dray, a urologist, criticizes Defense Secretary Pete Hegseth's recent plan to screen all service members aged 30 and older for testosterone, describing it as a modern echo of ancient…
    STAT · 1mo agoNegative↗ source
  • Nava Therapeutics raises $89 million to advance lipid nanoparticle platform targeting bone marrow, immune cells and kidney
    Nava Therapeutics, a stealth-mode biotech, announced it has secured $89 million in financing, according to a statement to Endpoints News. The capital will support development of a next generation…
    Endpoints · 1mo agoDealPositive↗ source
  • Roche stops two Huntington's disease gene-silencing programs after disappointing trial data
    Roche announced it is ending development of two gene‑silencing candidates aimed at Huntington's disease after data failed to meet expectations. The decision follows a recent readout that showed in…
    STAT · 1mo agoNegative↗ source
  • Statins and blood pressure drugs changing health risks of obesity, study suggests
    New research shows obesity patients over 40 are managing blood pressure and cholesterol as well as healthy-weight peers, but younger adults remain at risk.
    STAT · 1mo ago↗ source
  • FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease
    The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recu…
    fda press · 1mo ago↗ source
  • FDA Approves New Treatment That Uses Donor Immune Cells to Prevent Serious Complications in Blood Cancer Patients
    The U.S. Food and Drug Administration today approved TREGZI, the first regulatory T (Treg) cell-based immunotherapy for improving chronic graft-versus-host disease (GVHD)-free survival in adult pat…
    fda press · 1mo ago↗ source
  • Opinion: Short-sighted new student loan caps elevate chiropractic care over critical health care professions
    “This administrative logic is hopelessly out of touch with the empirical reality of American health care,” writes David S. Shapiro.
    STAT · 1mo ago↗ source
  • Biotech SPAC deal gets it to Nasdaq; AstraZeneca Phase 3 win in bone disease
    Plus news about Alebund Pharmaceuticals, Regenxbio and Neuphoria Therapeutics. 💸 A SPAC!: Talawar Therapeutics will land on the Nasdaq via a combination with blank-check company JATT II Acquisitio…
    Endpoints · 1mo ago↗ source
  • STAT+: Where ‘democracy met science,’ 50 years ago
    State Medicaid crackdowns strain home care, WHO leader on Ebola outbreak, and more health news
    STAT · 2mo ago↗ source