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13 stories matching this filter · ← front page

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STAT1156Endpoints875BioPharma Dive339BioSpace293Fierce Biotech277onclive165raps104globenewswire health73
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  • FDA clears Protagonist-Takeda therapy for rare blood cancer as Lilly moves to buy Merida for up to $2.88 billion
    The U.S. Food and Drug Administration has granted approval to a collaborative therapy from Protagonist and Takeda aimed at treating a rare form of blood cancer. This regulatory decision adds a new…
    raps · 7d agoDealPositive↗ source
  • FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation hurdles
    The U.S. Food and Drug Administration has granted approval to Ultragenyx's gene therapy aimed at treating a glycogen storage disorder. This marks a regulatory milestone for the company and the rare…
    raps · 18d agoApprovalPositive
fda medwatch65
fda recalls62
fda press38
858
Ophthalmology491
Pain468
Endocrinology & Metabolism465
Infectious Disease437
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  • Experts call for modernizing rare disease evidence models using FDA plausible mechanism framework
    A recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating. The authors suggest applying the FDA’s plausible mechanism framework,…
    raps · 18d agoNeutral↗ 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 · 21d agoRegulatoryNeutral↗ source
  • 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 · 1mo agoApprovalPositive↗ source
  • Phase III trial shows tebentafusp doubles five-year survival in untreated metastatic uveal melanoma
    A Phase III study of tebentafusp in patients with previously untreated metastatic uveal melanoma reported that five-year overall survival was roughly twice that of the control group. The trial als…
    onclive · 3mo agoPositive readoutPositive↗ source
  • MRI Features That Suggest Diffuse Midline Glioma Highlighted in Expert Discussion
    In a recent educational segment, Dr. Chong introduced the clinical scenario where patients with suspected brain tumors are first evaluated with imaging before tissue sampling. He then asked Dr. Sh…
    onclive · 3mo agoNeutral↗ source
  • Immunotherapy trials like PODIUM-303 are reshaping treatment for squamous cell anal cancer, says Dr Parikh
    Dr Aparna R. Parikh, an oncologist at Massachusetts General Hospital and Harvard professor, highlighted the growing feasibility of immunotherapy studies in squamous cell anal carcinoma (SCAC). She…
    onclive · 3mo agoPositive↗ 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 · 4mo 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 · 4mo 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 · 4mo 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 · 4mo ago↗ source