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  • RallyBio pursues second reverse merger, this time with cancer drug developer Avenzo Therapeutics
    RallyBio announced on Monday that it will merge with cancer-focused Avenzo Therapeutics in a reverse‑merger transaction. The combined entity will operate under the Avenzo Therapeutics name. The de…
    BioPharma Dive · 3mo agoDealNeutral↗ source
  • Hospitals grapple with patient requests for unvaccinated donor blood
    Some patients are now asking hospitals to provide blood that comes only from donors who have not received any COVID-19 vaccine, creating a new set of ethical and logistical questions. The demand r…
    STAT · 3mo agoNeutral↗ source
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  • #ASCO26: Lilly's plenaries; Incyte's frontline data in lymphoma
    Two plenaries related to Lilly: Researchers detailed findings from an investigator-sponsored trial using Lilly’s CDK4/6 inhibitor Verzenio in a rare cancer called dedifferentiated liposarcoma, show…
    Endpoints · 3mo agoNeutral↗ source
  • RevMed earns standing ovation at ASCO for pancreatic cancer data while Akeso faces caution
    At the American Society of Clinical Oncology meeting, RevMed captured audience attention with data on its pancreatic cancer therapy, earning a rare standing ovation. In contrast, presentations fro…
    STAT · 3mo agoNeutral↗ source
  • Former FDA oncology chief says political turmoil opens chance to reshape agency amid rising Chinese competition
    Rick Pazdur, who led oncology reviews at the FDA, told a STAT audience at the American Society of Clinical Oncology meeting that recent political appointments have caused significant disruption at…
    STAT · 3mo agoRegulatoryNeutral↗ source
  • Agios halts development of tebapivat for low-risk MDS after disappointing Phase 2b data
    Agios Pharmaceuticals announced it will discontinue development of its pyruvate kinase activator tebapivat for patients with low-risk myelodysplastic syndromes (MDS). The decision follows the relea…
    Endpoints · 3mo agoNegative↗ source
  • Allogene CEO David Chang to step down as Agios drug misses mid-stage trial results
    Allogene announced that its chief executive, co‑founder David Chang, will leave the role after an eight‑year tenure. Chang, who previously worked at Kite Pharma, will remain on the company’s board…
    BioPharma Dive · 3mo agoNegative readoutNeutral↗ source
  • Kenyan court halts U.S. plan for Ebola quarantine facility for Americans
    A Nairobi High Court on Friday ordered a stop to a U.S. proposal to build a quarantine center in Kenya for Americans who might have been exposed to a rare Ebola strain circulating in northeastern C…
    STAT · 3mo agoRegulatoryNegative↗ source
  • AbbVie receives FDA approval for ultra-rare blood cancer ADC acquired from ImmunoGen
    AbbVie announced that the U.S. Food and Drug Administration has granted approval for an antibody-drug conjugate targeting an ultra-rare, aggressive form of blood cancer. The therapy was obtained t…
    Endpoints · 3mo agoApprovalPositive↗ source
  • FDA Approves First Treatment for Chronic Hepatitis Delta Virus (HDV) Infection
    Today, the U.S. Food and Drug Administration approved Hepcludex (bulevirtide-gmod) injection to treat chronic hepatitis delta virus (HDV) infection in adults without cirrhosis (advanced liver scarr…
    fda press · 3mo agoApprovalPositive↗ source
  • AI tool guides Mayo clinicians to rapid treatment for newborn with ultra-rare disease
    An AI system called the Biomedical Data Translator was used by doctors at the Mayo Clinic to search for a therapy for a newborn with an ultra-rare disease. The infant, Jorie Kraus, was born with s…
    STAT · 3mo agoPositive↗ source
  • BioMarin's BMN-401 fails key endpoint in ENPP1 deficiency Phase 3 trial
    An experimental therapy, BMN 401, was tested in a Phase 3 study for ENPP1 deficiency, a rare disease. The drug missed one of its two co-primary endpoints, a result that regulators consider critical…
    BioPharma Dive · 3mo agoNegative readoutNegative↗ source
  • Relay Therapeutics reports promising Phase 2 results for zovegalisib; Eli Lilly leads R&D performance ranking
    Relay Therapeutics announced positive Phase 2 data for its experimental drug zovegalisib, which targets rare diseases that cause abnormal blood vessel development. The company said the results were…
    STAT · 3mo agoPositive readoutPositive↗ source
  • Relay Therapeutics' zovegalisib shows 60% response in mid‑stage trial for rare vascular anomalies
    Relay Therapeutics reported early data from a phase 2 trial of its experimental drug zovegalisib in patients with vascular anomalies, a group of rare disorders characterized by abnormal blood vesse…
    BioPharma Dive · 3mo agoPositive readoutPositive↗ source
  • Relay Therapeutics' zovegalisib meets efficacy endpoint in Phase 2 vascular anomalies trial
    Relay Therapeutics reported that its PI3Kα inhibitor zovegalisib achieved the predefined efficacy threshold in a Phase 2 study of a rare genetic vascular anomaly. The data readout exceeded Wall Str…
    Endpoints · 3mo agoPositive readoutPositive↗ source
  • BioMarin's Phase 3 trial of ENPP1 enzyme therapy fails to show benefit
    BioMarin reported that its late-stage study of an enzyme replacement therapy for ENPP1 deficiency did not meet its primary efficacy endpoints. The trial, which enrolled patients lacking the ENPP1 e…
    Endpoints · 3mo agoNegative readoutNegative↗ source
  • Amgen defends Tavneos as FDA, European regulators and Japanese doctors raise safety concerns
    Amgen said it continues to support its rare-disease therapy Tavneos despite recent safety alerts. The U.S. FDA has indicated it wants the drug removed from the market after a report of 20 deaths l…
    Endpoints · 3mo agoRegulatoryNegative↗ source
  • FDA leadership overhaul follows firing of acting CDER director; 20 deaths linked to Amgen’s Tavneos in Japan
    The FDA announced another leadership change, removing acting directors of its two main drug review centers. Tracy Beth Høeg, acting head of the Center for Drug Evaluation and Research (CDER), poste…
    BioPharma Dive · 3mo agoRegulatoryNegative↗ source
  • Trump administration cuts childhood vaccine schedule, citing personal autonomy
    The opinion argues that the concept of patient autonomy does not apply to childhood vaccination because infants cannot refuse shots; the decision rests with parents. In January, the Department of…
    STAT · 3mo agoRegulatoryNegative↗ source
  • Goodman urges five reforms to restore trust in CDC vaccine advisory committee
    Jesse Goodman, a former director of the FDA's Center for Biologics Evaluation and Research and former chief scientist, wrote an opinion piece calling for major changes to the CDC's Advisory Committ…
    STAT · 3mo agoRegulatoryNegative↗ source
  • Texas launches clinic to support individuals seeking detransition services
    Texas health officials announced the opening of a clinic dedicated to people who want to reverse a gender transition, marking a new service in the state. The facility will provide counseling, medi…
    STAT · 3mo agoNeutral↗ source
  • Americans in Congo May Have Been Exposed to Ebola; One Shows Possible Symptoms, Tests Pending
    Several Americans traveling in the Democratic Republic of the Congo are believed to have had high risk exposure to suspected Ebola cases, according to sources familiar with the situation. At least…
    STAT · 3mo agoNeutral↗ source
  • Hantavirus outbreak on cruise sparks Covid-era conspiracy chatter, officials say risk remains low
    A rare strain of hantavirus infected several passengers on a cruise ship, prompting health authorities to investigate the outbreak and issue public updates. Officials emphasized that, unlike Covid…
    STAT · 3mo agoNeutral↗ source
  • FDA Commissioner Marty Makary Resigns, Prompting Concerns Over Agency Direction
    FDA Commissioner Marty Makary stepped down on Tuesday, leaving the agency without a permanent leader as it already faces turnover in its drug and biologics divisions. Makary had recently appeared…
    STAT · 3mo agoNegative↗ source
  • Biotech execs push for Rick Pazdur as FDA chief while Takeda announces 4,500 layoffs
    Hundreds of biotechnology executives, investors and patient advocates have signed a petition urging President Trump to nominate Rick Pazdur as the next FDA commissioner. Pazdur, a longtime FDA vet…
    BioPharma Dive · 3mo agoLayoffsNeutral↗ source