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  • 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
  • FDA rejects Hengrui-Elevar liver cancer drug combo for third time
    For the third time in about two years, the FDA has cited manufacturing deficiencies in rejecting a drug combination from Jiangsu Hengrui Pharmaceuticals and Elevar Therapeutics intended to treat th…
    Endpoints · 20d ago↗ source
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FDA Approves New Indication for Tzield (teplizumab) for Certain Pediatric Patients with Recently Diagnosed Stage 3 Type 1 Diabetes
On June 12, 2026, the U.S. Food and Drug Administration granted accelerated approval to Tzield (teplizumab) for a new indication, to delay the decline of insulin production in pediatric patients ag…
fda press · 1mo ago↗ source
  • City Therapeutics gets $99M to gather data on three RNAi medicines
    City Therapeutics, the next-gen RNAi startup founded and led by former Alnylam executives, has collected $99.5 million to fund the clinical development of its first three candidates and expand into…
    Endpoints · 1mo ago↗ source
  • GSK inks multi-target pact with Engitix; Sangamo considers strategic options
    Plus news about Pfizer's latest label expansion. 🤝 GSK inks multi-target liver pact with Engitix: London-based Engitix will use disease models based on its extracellular matrix technology to ident…
    Endpoints · 1mo ago↗ source