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24 stories matching this filter · ← front page

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  • FDA approves Zanvastro, first therapy targeting protein buildup in Alexander disease
    The U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in both pediatric and adult patients. Zanvastro is the first FDA-approve…
    fda press · 8d agoApprovalPositive↗ source
  • 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 · 12d agoDealPositive↗ source
73
fda recalls71
fda medwatch62
fda press38
883
Ophthalmology520
Pain501
Endocrinology & Metabolism494
Infectious Disease456
  • FDA approves Mimrylo, first hepcidin-mimicking drug for polycythemia vera
    The U.S. Food and Drug Administration announced approval of Mimrylo (rusfertide) for adult patients with polycythemia vera, a rare blood disorder that causes excess red blood cells. The drug mimic…
    fda press · 14d agoApprovalPositive↗ source
  • FDA greenlights first oral therapy for adult dermatomyositis
    The U.S. Food and Drug Administration has approved Lisraya (brepocitinib) tablets as a treatment for adults with dermatomyositis. Dermatomyositis is a rare autoimmune condition that attacks muscle…
    fda press · 15d agoApprovalPositive↗ source
  • FDA approves Rasonque, first-in-class RAS inhibitor for metastatic pancreatic cancer
    The U.S. Food and Drug Administration has approved Rasonque (daraxonrasib), a RAS inhibitor, for the treatment of metastatic pancreatic adenocarcinoma. Rasonque is a tablet taken once daily that t…
    fda press · 17d agoApprovalPositive↗ 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 · 23d agoApprovalPositive↗ source
  • 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 · 23d agoNeutral↗ source
  • 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 · 23d 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 · 26d 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
  • 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 · 1mo agoApprovalPositive↗ source
  • FDA Grants First Gene-Therapy Approval for Children 2+ with Sickle Cell Disease
    The U.S. Food and Drug Administration issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged two years and older who have sickle cell disease with recurrent vaso-oc…
    fda press · 2mo agoApprovalPositive↗ source
  • FDA approves Tregzi, first T‑reg cell therapy to lower chronic GVHD risk in blood‑cancer transplants
    The FDA announced approval of Tregzi, a regulatory T‑cell based immunotherapy designed to improve chronic graft‑versus‑host disease‑free survival for adults with blood cancers undergoing allogeneic…
    fda press · 2mo agoApprovalPositive↗ source
  • FDA picks seven firms for PreCheck pilot to boost U.S. drug manufacturing
    The FDA announced on June 29, 2026 that seven companies have been chosen for its PreCheck Pilot Program, an initiative aimed at strengthening U.S. drug manufacturing and supply chain resilience. T…
    fda press · 2mo agoRegulatoryPositive↗ source
  • FDA releases draft guidance to speed cell and gene therapy approvals
    The FDA announced a draft guidance aimed at helping developers bring gene therapies to patients more quickly. It encourages use of publicly available scientific data and existing platform knowledge…
    fda press · 3mo agoRegulatoryNeutral↗ 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 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 · 4mo 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 · 4mo 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 · 4mo agoPositive↗ source
  • MimiVax and Roswell Park start Phase 2 trial of SurVaxM with temozolomide for metastatic neuroendocrine tumors
    MimiVax Inc. and Roswell Park Comprehensive Cancer Center announced the launch of a randomized Phase 2 study evaluating SurVaxM in combination with temozolomide for patients with progressing metast…
    onclive · 4mo agoCRLNeutral↗ source
  • FDA approves Zenocutuzumab for NRG1 fusion-positive advanced cholangiocarcinoma
    The U.S. Food and Drug Administration has granted approval for zenocutuzumab-zbco, marketed as Bizengri, to treat adult patients with NRG1 fusion‑positive advanced, unresectable or metastatic chola…
    onclive · 4mo agoApprovalPositive↗ source
  • Study Finds Screening Gap for HTLV-1 Linked Leukemia in US Caribbean-Born Communities
    Researchers at Sylvester Comprehensive Cancer Center have highlighted a gap in screening for adult T‑cell leukemia/lymphoma (ATLL) that could be addressed by preventing mother‑to‑child transmission…
    onclive · 4mo agoPositive↗ source
  • Experts outline PET-CT, biopsy and mutation testing to guide therapy in relapsed CLL
    In a recent discussion, clinicians described the workup for patients with progressive chronic lymphocytic leukemia (CLL). They emphasized using PET‑CT imaging and excisional lymph node biopsy when…
    onclive · 4mo agoNeutral↗ source
  • Daraxonrasib uniquely targets active KRAS in pancreatic cancer, says MD Anderson expert
    Dr Shubham Pant, a professor in the Department of Gastrointestinal Medical Oncology at MD Anderson Cancer Center, explained the mechanism of daraxonrasib in a recent commentary. The oral, potent R…
    onclive · 4mo agoPositive↗ source