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6 stories matching this filter · ← front page

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STAT825Endpoints630BioPharma Dive240onclive165Fierce Biotech91BioSpace90raps32globenewswire health
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allOncology369Immunology179Rare Disease160Hematology91Cardiology81Pulmonology69Ophthalmology
  • 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 · 1d 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 · 3d agoNeutral↗ source
28
fda recalls22
fda press21
fda medwatch17
60
Vaccines49
Hepatology44
Psychiatry & CNS37
Dermatology35
Pain33
  • 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 · 6d 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 · 6d 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 · 6d ago↗ source
  • FDA approves Regeneron's hearing loss gene therapy Otarmeni
    Regeneron has won the first-ever FDA approval for a gene therapy to treat a rare type of inherited hearing loss, and the company plans to offer the treatment for free in the US, though that ...
    Endpoints · 3mo ago↗ source