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15 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 · 23h agoApprovalPositive↗ source
  • Saudi FDA launches program to accelerate rare-disease drug development
    The Saudi Food and Drug Authority (SFDA) announced a new initiative called the New Accelerator Program for Drugs in Rare Diseases (NADR). The program is designed to speed up the development, evalu…
    raps · 3d agoRegulatoryNeutral↗ source
73
fda recalls47
fda medwatch38
fda press28
899
Psychiatry & CNS712
Ophthalmology421
Endocrinology & Metabolism382
Pain375
Infectious Disease356
  • FDA approves first drug for type 1 narcolepsy and greenlights mRNA flu vaccine; AstraZeneca and BMS deny merger talks
    The U.S. Food and Drug Administration has granted approval for the first therapy specifically targeting type 1 narcolepsy, marking a milestone for patients with this rare sleep disorder. In a sepa…
    raps · 14d agoApprovalPositive↗ source
  • FDA approves Orzeyful, first drug to treat all symptoms of narcolepsy type 1
    The U.S. Food and Drug Administration announced on August 5, 2026 that it has approved Orzeyful (oveporexton) tablets for adults with narcolepsy type 1. Orzeyful is the first therapy cleared to tr…
    fda press · 15d agoApprovalPositive↗ 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 · 1mo 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
  • 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 · 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 · 3mo ago↗ source
  • Dr Parikh on the Feasibility and Importance of Immunotherapy Trials in Anal Cancer
    Aparna R. Parikh, MD, discusses how trials like PODIUM-303 are reshaping the treatment paradigm for squamous cell anal carcinomas.
    onclive · 3mo ago↗ source
  • MimiVax and Roswell Park Open Phase 2 Trial of SurVaxM for Patients with Metastatic Neuroendocrine Tumors
    Roswell Park Comprehensive Cancer Center launched a phase 2 trial evaluating SurVaxM plus temozolomide in progressing neuroendocrine tumors.
    onclive · 3mo ago↗ source
  • FDA Approves Zenocutuzumab for NRG1 Fusion+ Cholangiocarcinoma
    onclive · 3mo ago↗ source
  • Sylvester Comprehensive Cancer Center Identifies a Screening Gap to Prevent a Type of Leukemia in the U.S.
    Researchers at Sylvester identified populations in the US where this cancer may be largely preventable by addressing mother‑to‑child transmission of the virus.
    onclive · 3mo ago↗ source
  • Disease Assessment and Mutational Testing in Progressive CLL
    Experts discuss relapsed CLL workup, using PET-CT and lymph node biopsy to rule out Richter’s, plus resistance testing to guide next therapy.
    onclive · 3mo ago↗ source
  • Dr Pant on the Mechanism of Action of Daraxonrasib in PDAC
    Shubham Pant, MD, MBBS, explains the unique "on-state" binding mechanism of daraxonrasib.
    onclive · 3mo ago↗ source