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  • FDA lifts opposition to Huntington’s disease gene therapy, clearing path for development
    The Readout Loud podcast this week highlighted a regulatory shift for a gene therapy aimed at Huntington’s disease. The Food and Drug Administration reversed its earlier opposition, removing a key…
    STAT · 2mo agoRegulatoryPositive↗ source
  • UniQure plans to file Huntington’s disease gene therapy with FDA after leadership shift eases data requirements
    UniQure, a Dutch biotech, announced it will seek FDA approval for its gene therapy targeting Huntington’s disease later this summer. The company said FDA staff indicated that three years of data f…
    BioPharma Dive · 2mo agoRegulatoryNeutral↗ source
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  • FDA says it will maintain Makary’s focus on clinical trials, rare diseases and China despite leadership exits
    The Food and Drug Administration announced that, even after the recent resignations of Commissioner Marty Makary and the heads of its drug and biologics centers, it will keep pursuing the policy ag…
    Endpoints · 2mo agoRegulatoryNeutral↗ source
  • Covid vaccine linked to 24% drop in overall cardiac events, study of 1 million veterans shows
    A new analysis of more than one million U.S. veterans indicates that receiving a Covid‑19 vaccine is associated with a substantial reduction in heart‑related problems. Researchers observed a roughl…
    STAT · 2mo agoPositive readoutPositive↗ source
  • Lilly reports early positive results for new JAK2 inhibitor in hard-to-treat myelofibrosis
    Lilly has incorporated a JAK2‑targeting drug originally discovered by Ajax Therapeutics after its recent acquisition. The experimental pill is intended for patients with myelofibrosis, a difficult‑…
    BioPharma Dive · 2mo agoPositive readoutPositive↗ source
  • FDA approves Sanofi's teplizumab for children with stage 3 type 1 diabetes
    The FDA announced approval of teplizumab, a Sanofi developed therapy, for children aged 8 and older who have stage 3 type 1 diabetes. The decision came through the agency’s accelerated review path…
    STAT · 2mo agoApprovalPositive↗ source
  • WHO chief warns of worsening Ebola outbreak in DRC and Uganda after on-site visit
    The World Health Organization's director‑general said he is "really worried" after returning from a second trip to the Ebola‑affected regions of the Democratic Republic of the Congo and Uganda. He…
    STAT · 2mo agoNegative↗ source
  • Amgen seeks FDA review of Tavneos to determine continued market availability
    Amgen has formally asked the U.S. Food and Drug Administration to re-examine the data supporting its drug Tavneos (avacopan). Tavneos is indicated for a rare form of vasculitis and the request sig…
    Endpoints · 2mo agoRegulatoryNeutral↗ source
  • Blood Cancer United acquires remaining supply of discontinued investigational drug Luvelta
    Blood Cancer United, a nonprofit focused on blood cancers, is purchasing the remaining inventory of Luvelta, an investigational cancer drug that has been discontinued. The acquisition aims to pres…
    STAT · 2mo agoNeutral↗ source
  • Biopharma exec pay reveals quirky perks like private jets and wildfire relief grants
    A recent review of thousands of biopharma C‑suite compensation packages found that most line items blend into the massive totals, but a few stand out for their unusual nature. Among the highlighte…
    Endpoints · 2mo agoNeutral↗ source
  • J&J reports positive Phase 2/3 results for Imaavy in rare autoimmune anemia, paving way for label expansion
    Johnson & Johnson announced that its drug Imaavy, already approved for myasthenia gravis, produced durable hemoglobin improvements in a Phase 2/3 trial for warm autoimmune hemolytic anemia, a rare…
    BioPharma Dive · 2mo agoPositive readoutPositive↗ source
  • Novartis' RNA drug del-brax hits primary endpoint in early-stage FSHD trial
    Novartis reported that its antisense oligonucleotide conjugate, del-brax, achieved the primary biomarker endpoint in a Phase 1/2 study of patients with facioscapulohumeral muscular dystrophy (FSHD)…
    BioPharma Dive · 2mo agoPositive readoutPositive↗ source
  • Nonprofit Blood Cancer United purchases remaining Luvelta supplies to keep compassionate access for children
    Blood Cancer United, formerly the Leukemia & Lymphoma Society, announced it will purchase the remaining inventory of Luvelta, an investigational antibody‑drug conjugate that had been discontinued.…
    STAT · 2mo agoDealPositive↗ source
  • CDC report finds rise in alcohol use among pregnant women since 2020
    A new CDC analysis shows that the proportion of pregnant adults who reported drinking in the past month increased after 2020. The survey data indicate that about 15% of pregnant women reported cur…
    STAT · 2mo agoNegative↗ source
  • Sensorion drops OTOF gene therapy to focus on GJB2-targeted hearing loss candidate after Regeneron approval
    Sensorion said it is ending development of its gene therapy SENS-501 for OTOF related hearing loss after a strategic review. The review concluded that the competitive landscape had changed signific…
    BioPharma Dive · 2mo agoNeutral↗ source
  • Sanofi halts Phase 3 trial of riliprubart in CIDP after interim analysis shows unlikely efficacy
    Sanofi announced Wednesday that it is stopping the Phase 3 MOBILIZE study of its experimental anti‑inflammatory drug riliprubart in patients with chronic inflammatory demyelinating polyneuropathy (…
    BioPharma Dive · 2mo agoNegative readoutNegative↗ source
  • Beren Therapeutics secures $300 million to fund rare disease drug launch ahead of FDA review
    Beren Therapeutics announced it has raised $300 million in new financing to support the development and launch of its rare disease therapy. The funding arrives as the company prepares for an FDA d…
    Endpoints · 2mo agoDealPositive↗ source
  • Sanofi halts rare disease trial as Parabilis launches record biotech IPO amid European pricing debate
    Sanofi announced it is stopping a Phase 3 trial of its experimental drug riliprubart in a rare autoimmune disease after an interim analysis showed the treatment did not work. The pause adds to rec…
    STAT · 2mo agoNeutral↗ source
  • Sanofi halts Phase 3 trial of riliprubart in rare neurological disorder
    Sanofi announced it is ending the Phase 3 study of its antibody drug riliprubart aimed at treating a rare neurological disease. The decision follows a recommendation from an independent data monit…
    Endpoints · 3mo agoNegative readoutNegative↗ source
  • DOJ announces second settlement on youth gender-affirming care amid broader health news
    The Department of Justice disclosed that it has reached a second settlement with parties involved in providing gender-affirming care to minors. The agreement, announced in a recent newsletter, buil…
    STAT · 3mo agoRegulatoryNeutral↗ source
  • Grace Science warns FDA bottleneck threatens ultra-rare gene therapy progress
    Grace Science, a biotech focused on ultra-rare diseases, has been developing a gene-therapy candidate that appeared close to regulatory approval. The company, co-founded by Nobel laureate Carolyn…
    Endpoints · 3mo agoRegulatoryNegative↗ source
  • J&J to buy Firefly Bio for $1 billion to boost KRAS inhibitor program
    J&J announced Monday it will acquire biotech startup Firefly Bio for $1 billion, aiming to broaden its KRAS inhibitor portfolio. The move follows recent data from Revolution Medicines showing a KR…
    STAT · 3mo agoDealPositive↗ source
  • Congo Ebola frontline health workers face low pay and exhaustion amid outbreak
    Dr. Richard Lokudu, medical director of Mongbwalu General Referral Hospital, says he and his colleagues are receiving almost no compensation while treating patients in the current Ebola outbreak.…
    STAT · 3mo agoNegative↗ source
  • Growing Amish population poses unique public health challenges, says STAT opinion piece
    The First Opinion podcast explored how the expanding Amish community impacts public health, noting that the group may approach 2 million people in the United States by 2075. While some political fi…
    STAT · 3mo agoNeutral↗ source
  • HHS Office of Human Research Protections faces staff shortages, jeopardizing participant safety oversight
    In 2021 the Office of Human Research Protections (OHRP) received a report of a participant suicide during a depression and mobility study at the New York State Psychiatric Institute. The incident p…
    STAT · 3mo agoRegulatoryNegative↗ source