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42 stories matching this filter · ← front page

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  • FDA and Capricor clash over Duchenne cell therapy efficacy at advisory committee
    At an FDA advisory committee meeting, the agency and Capricor Therapeutics presented conflicting views on the efficacy data for the company's experimental cell therapy aimed at Duchenne muscular dy…
    Endpoints · 2d agoRegulatoryNegative↗ source
  • Updated: AstraZeneca foresees blockbuster sales for Claudin 18.2 ADC, but Ultomiris flops in rare disease
    AstraZeneca said Monday that a key late-stage trial of its Claudin 18.2-targeting ADC in stomach cancer had succeeded, albeit with a caveat — but another Phase 3 trial of Ultomiris yielded subpar r…
    Endpoints · 4d agoNeutral↗ source
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570
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Endocrinology & Metabolism290
Ophthalmology255
Infectious Disease253
Nephrology238
  • Amgen submits new analysis to FDA to defend continued availability of Tavneos
    Amgen has provided the FDA with a fresh analysis concerning its rare-disease therapy Tavneos, aiming to address the agency’s concerns. The company says the independent reevaluation supports the dr…
    Endpoints · 7d agoRegulatoryNeutral↗ source
  • Ipsen's Bylvay fails Phase 3 trial in children with biliary atresia
    Ipsen announced that its experimental therapy Bylvay did not meet its primary endpoint in a Phase 3 trial targeting biliary atresia in infants. Biliary atresia is a rare pediatric liver disorder i…
    Endpoints · 7d agoNegative readoutNegative↗ source
  • CHMP recommends Minoryx’s ALD drug, rejects Zevra’s Meplyffa
    Minoryx Therapeutics finally scored backing from EU regulators for its neurological drug that’s faced multiple hurdles. Meanwhile, Zevra’s rare disease drug got a setback in Europe almost two years…
    Endpoints · 7d ago↗ source
  • Broad Institute launches Center for Therapeutic Genetics for rare diseases
    The Broad Institute of MIT and Harvard is teaming up with Boston Children’s Hospital and The Jackson Laboratory to launch a new center focused on genetic treatments for rare diseases, the triumvira…
    Endpoints · 10d ago↗ source
  • ARPA-H bookmarks $160M for custom gene editing treatments. Is it enough?
    After an infant last year was saved from a deadly disease thanks to a bespoke gene editing therapy that fixed an incredibly rare mutation in his DNA, the US government is hoping ...
    Endpoints · 21d ago↗ source
  • Early Alert: Convenience Kits Issue from Medline
    Certain BD ChloraPrep Applicators in Convenience Kits manufactured by Medline may not be sterile.
    fda medwatch · 22d 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 · 29d 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
  • FDA Selects Seven Participants for PreCheck Pilot Program to Advance U.S. Drug Manufacturing
    FDA announces the selection of seven companies to participate in the FDA PreCheck Pilot Program, a new initiative designed to boost American global competitiveness, strengthen domestic drug manufac…
    fda press · 1mo ago↗ source
  • Endpoints News History: 10 Years, Bootstrap to FT
    Ten years of Endpoints News: no investors, a 50/50 split, profitable from day one. Co-founders Arsalan Arif and John Carroll on how the partnership held.
    Endpoints · 1mo ago↗ source
  • FDA plans to keep Makary priorities on trials, China, deputy says
    PHILADELPHIA – Following the recent departures of FDA Commissioner Marty Makary and both of his center directors for drugs and biologics, the Trump administration will continue pushing their priori…
    Endpoints · 1mo ago↗ source
  • Amgen and the FDA want two different outcomes for Tavneos. What will patients do?
    Amgen has asked the FDA to review evidence that its drug Tavneos (avacopan), which treats a rare form of vasculitis, is worth keeping on the market, as the future of the drug remains in flux. ...
    Endpoints · 1mo ago↗ source
  • Biopharma's pay oddities of 2025: Executive perks and eccentricities
    In combing through thousands of C-suite pay packages worth billions of dollars, it’s rare for a line item to stick out. Every once in a while, a biopharma company discloses perks that go beyond the…
    Endpoints · 1mo ago↗ source
  • Rare disease biotech Beren adds $300M from new financings
    Anxiously awaiting an FDA decision this fall, Beren Therapeutics has added $300 million in new capital to help launch its rare disease drug. The biotech announced Wednesday that it raised $135 ...
    Endpoints · 1mo ago↗ source
  • Sanofi to mull riliprubart’s future after stopping late-stage nerve disease study
    Sanofi is ending a Phase 3 trial of its antibody drug in a rare neurological disorder after an independent committee said the study was unlikely to show sufficient efficacy. The broader future of t…
    Endpoints · 1mo ago↗ source
  • Grace Science says FDA bottleneck jeopardizes rare disease therapy
    Grace Science appeared to be one of the few companies fighting ultra-rare diseases whose gene therapy candidate might make it across the finish line. Co-founded by Nobel laureate Carolyn Bertozzi,…
    Endpoints · 1mo ago↗ source
  • Industry seeks more clarity on commissioner's voucher program
    The pharma industry and rare disease advocates voiced concerns about the transparency and fairness of the Commissioner's National Priority Voucher pilot program. The program faces an uncertain futu…
    Endpoints · 1mo ago↗ source
  • Acting FDA commissioner pledges to prevent political interference in rare disease meeting
    Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas made it clear he's headed in a different…
    Endpoints · 1mo ago↗ source
  • Travere pays Everest $112M upfront to grow rare kidney disease pipeline
    Travere Therapeutics is enlisting the help of China-based Everest Medicines to bolster its rare kidney disease pipeline beyond its approved medicine Filspari. The San Diego biotech is doling out $1…
    Endpoints · 1mo ago↗ source
  • FDA Issues Draft Guidance to Help Accelerate Cell and Gene Therapies for Patients
    The U.S. Food and Drug Administration (FDA) today issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific a…
    fda press · 1mo ago↗ source
  • #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 · 2mo ago↗ source
  • Agios ends development of tebapivat in low-risk MDS following subpar results
    Agios Pharmaceuticals has said it will stop developing its rare blood disorder drug for certain patients with myelodysplastic syndromes (MDS) due to disappointing Phase 2b data. The biotech’s pyruv…
    Endpoints · 2mo ago↗ source
  • AbbVie clinches FDA approval for ultra-rare blood cancer ADC from ImmunoGen purchase
    AbbVie said the FDA has approved an antibody-drug conjugate it obtained as part of its $10.1 billion buyout of ImmunoGen for an ultra-rare but aggressive form of blood cancer. On Wednesday, the ...
    Endpoints · 2mo ago↗ source