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14 stories matching this filter · ← front page

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  • FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type Ia
    The U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen…
    fda press · 19h agoApprovalPositive↗ source
  • Optimal Balance Pharmacy Issues Voluntary Nationwide Recall of Certain Lots of Compounded Glutathione 200 mg/mL Multi-Dose Vials Due to Elevated Endotoxin Levels
    FOR IMMEDIATE RELEASE – August 19, 2026 – Houston, Texas, Optimal Balance Pharmacy is voluntarily recalling one lot of Compounded Glutathione 200 MG/ML 30 ML Multi-Dose Vials to the patient level.…
    fda recalls · 1d ago↗ source
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fda recalls47
fda medwatch37
fda press28
899
Psychiatry & CNS712
Ophthalmology421
Endocrinology & Metabolism380
Pain373
Infectious Disease353
  • Intellia finds genetic marker linked to liver enzyme spikes in CRISPR ATTR amyloidosis trial
    Intellia Therapeutics announced that analysis of its ongoing CRISPR gene-editing trial for transthyretin (ATTR) amyloidosis has identified a common genetic variant among participants who experience…
    Endpoints · 14d agoNegative readoutNegative↗ 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 · 27d agoNegative readoutNegative↗ source
  • FDA rejects Hengrui-Elevar liver cancer drug combo for third time over manufacturing issues
    FDA has again turned down a liver cancer drug combination from Jiangsu Hengrui Pharmaceuticals and Elevar Therapeutics. This is the third rejection in roughly two years, with the agency citing per…
    Endpoints · 1mo agoRegulatoryNegative↗ source
  • 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 · 2mo 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 · 2mo 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 · 2mo ago↗ source
  • #ASCO26: AstraZeneca's triplet combo succeeds in liver cancer subgroup
    CHICAGO — AstraZeneca on Monday reported data from a new study showing the combination of a targeted treatment and two immunotherapies can benefit patients with certain kinds of liver cancer. Indiv…
    Endpoints · 2mo ago↗ 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 · 2mo ago↗ source
  • Amgen stands by rare disease drug Tavneos amid Japan liver toxicity report
    The FDA wants it off the market. European regulators are investigating data-integrity concerns. And now, a pharma company selling the drug in Japan is warning doctors against writing new prescripti…
    Endpoints · 3mo ago↗ source
  • Rezdiffra is exceeding Wall Street's expectations as the MASH market takes shape
    It seems there’s room for two in the MASH market. When Novo Nordisk’s blockbuster weight loss drug Wegovy won approval in August 2025 to treat the fatty liver disease, some investors thought it wou…
    Endpoints · 3mo ago↗ source
  • Intercept's next-gen FXR agonist fails Phase 2 in latest setback for Italian-owned pharma
    Intercept Pharmaceuticals' attempt at creating a next-generation FXR agonist for certain forms of liver inflammation appears to have ended. The drug developer's INT-787 showed "no clear evidence of…
    Endpoints · 3mo ago↗ source
  • Madrigal extends MASH deal streak with Arrowhead drug that J&J didn't want
    Madrigal Pharmaceuticals has made its fourth MASH deal in 10 months, further signaling its ambitions to be not just the first drugmaker for the fatty liver disease, but also the owner of the field'…
    Endpoints · 3mo ago↗ source