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247 stories matching this filter · ← front page

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  • Ionis receives FDA approval for Zanvastro, the first treatment for Alexander disease, as Pfizer partners on new ADC program
    The U.S. Food and Drug Administration approved Zanvastro, marking the first approved medicine for the rare and often fatal neurological disorder Alexander disease. Developed by Ionis Pharmaceutical…
    BioPharma Dive · 3d agoApprovalPositive↗ source
  • Licensing deals with Chinese biotech stay strong as big pharma pours $550M+ into cancer and obesity pipelines
    Western drug developers continue to sign licensing agreements with Chinese biotechnology firms at a steady pace, according to recent data visualizations. More than 100 such deals have been announc…
    BioPharma Dive · 3d agoDealNeutral↗ source
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Infectious Disease437
  • Ionis secures FDA approval for zilganersen, its first solo neurology therapy for Alexander disease
    Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has granted approval to its investigational RNA‑based drug zilganersen for the treatment of Alexander disease, a rare neur…
    Endpoints · 3d agoApprovalPositive↗ source
  • MassBio and SCbio select 10 life science startups for Drive accelerator
    MassBio and SCbio announced that ten early‑stage life‑science companies have been chosen for the fall cohorts of the Drive accelerator program. The selection includes five biotech firms and five te…
    BioSpace · 3d agoPositive↗ source
  • CLINUVEL to cut up to 20% of staff and move headquarters to New York
    CLINUVEL announced a strategic reorganization aimed at focusing resources on the U.S. market. The company will reduce its global workforce by 10% to 20%, affecting up to 21 of its 104 employees.…
    BioSpace · 3d agoLayoffsNegative↗ source
  • Ultragenyx’s Angelman therapy fails Phase 3, stock drops over 43% and company announces strategic review
    Ultragenyx reported that its investigational Angelman syndrome drug apazunersen did not meet the primary cognitive endpoints in a Phase 3 trial. The lack of efficacy means the therapy will not prov…
    BioSpace · 4d agoNegative readoutNegative↗ source
  • FDA approves Ionis’ Zanvastro as first disease-modifying therapy for Alexander disease
    The U.S. Food and Drug Administration has granted approval to Ionis Pharmaceuticals' antisense oligonucleotide Zanvastro, the first therapy that modifies disease progression for Alexander disease.…
    BioSpace · 3d agoApprovalPositive↗ source
  • Ultragenyx to consider major cost cuts after Angelman drug fails trial
    Ultragenyx announced that its experimental therapy GTX-102 (apazunersen) for Angelman syndrome failed to meet primary and secondary endpoints in a Phase 3 study. The company said there were no dif…
    BioPharma Dive · 4d agoNegative readoutNegative↗ source
  • Revolution Medicines' Rasonque shows 42% response and 16-month survival in NSCLC subgroup
    Data from a subgroup of an earlier trial, described by Truist Securities as the most relevant to Revolution Medicines' ongoing Phase 3 study, reported a 42% overall response rate and a median overa…
    BioSpace · 4d agoPositive readoutPositive↗ source
  • Superluminal Medicines raises $60 million to advance AI-designed MC4R drug for rare obesity forms
    Superluminal Medicines, an AI-driven drug discovery startup, announced a $60 million financing round aimed at moving its first candidate toward clinical testing. The candidate targets the MC4R G p…
    BioPharma Dive · 4d agoDealPositive↗ source
  • ARPA-H allocates up to $125 million to accelerate personalized RNA therapy manufacturing
    ARPA-H announced a new program, called GIVE, that will provide up to $125 million in research funding to develop faster, decentralized manufacturing methods for individualized RNA‑based genetic med…
    BioSpace · 4d agoRegulatoryPositive↗ source
  • Senior FDA official overrides reviewers to grant Replimune accelerated approval for melanoma drug Tudriqev
    FDA's primary review team recommended rejecting Replimune's melanoma therapy Tudriqev, saying the company had not adequately addressed key deficiencies noted in earlier complete response letters.…
    BioSpace · 4d agoRegulatoryNeutral↗ source
  • Ultragenyx's Angelman syndrome drug fails Phase 3 trial, halting development
    Ultragenyx reported that its experimental antisense oligonucleotide designed for Angelman syndrome did not meet primary endpoints in a Phase 3 study. The trial, which enrolled patients with the ra…
    Endpoints · 4d agoNegative readoutNegative↗ source
  • FDA advisory panels for Capricor and Replimune highlight need for rare-disease specialist input
    Recent FDA advisory committee meetings reviewing therapies from Capricor Therapeutics and Replimune produced markedly different outcomes, underscoring how the agency’s reliance on specialist input…
    BioSpace · 4d agoRegulatoryNegative↗ source
  • Novartis and BMS pause CAR‑T trials after patient deaths while Revolution Medicines gains early FDA approval for pancreatic cancer drug
    Novartis has placed several trials of its CAR‑T therapy rapcabtagene autoleucel on hold after three patients receiving the treatment died. Bristol Myers Squibb also voluntarily froze its autoimmun…
    BioSpace · 5d agoRegulatoryNeutral↗ source
  • FDA to tighten oversight of foreign clinical trial sites as China trial volume soars
    FDA officials wrote an editorial stating that inspection access is not an administrative footnote and announced plans to expand oversight of foreign clinical trial sites. The initiative follows th…
    BioSpace · 4d agoRegulatoryNeutral↗ source
  • UniQure submits FDA and UK applications for first Huntington’s disease gene therapy
    UniQure has filed a biologics license application (BLA) with the U.S. FDA and a separate submission with the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its experimental gene…
    BioSpace · 5d agoRegulatoryNeutral↗ source
  • UK regulator suspends new patients from receiving Tavneos (Avacopan Vifor) amid data concerns
    The UK Medicines and Healthcare products Regulatory Agency (MHRA) has decided to stop prescribing Tavneos, known locally as Avacopan Vifor, to any new patients. The decision follows the regulator’…
    BioSpace · 5d agoRegulatoryNegative↗ source
  • Novartis and BMS pause CAR-T trials after fatal immune syndrome cases
    Novartis has halted several immunology and neurology studies of its CAR T candidate rapcabtagene autoleucel after three patients experienced fatal immune effector cell‑associated hemophagocytic syn…
    BioSpace · 6d agoRegulatoryNegative↗ source
  • GSK to start Phase 3 trial of mRNA flu vaccine after strong mid-stage results
    GSK announced it will begin a Phase 3 trial of its mRNA-based influenza vaccine, with enrollment slated to start in September. In a Phase 2 study of 971 adults, the investigational vaccine, which…
    BioSpace · 6d agoPositive readoutPositive↗ source
  • Nine midsize biopharma firms join Trump MFN drug pricing program, covering 89% of branded market
    Nine midsize biopharma companies have agreed to voluntarily participate in the Trump administration's Most Favored Nation drug pricing program, adding to the 17 large pharmaceutical firms that prev…
    BioSpace · 6d agoRegulatoryPositive↗ source
  • FDA clears Takeda and Protagonist's rusfertide (Mimrylo) for polycythemia vera, opening potential $2 billion market
    The Food and Drug Administration has granted clearance to rusfertide, sold under the brand name Mimrylo, for the treatment of polycythemia vera, a rare blood disorder that causes excess red blood c…
    BioPharma Dive · 7d agoApprovalPositive↗ source
  • Biomarin nabs big royalties in achondroplasia patent settlement with Ascendis
    Biomarin has granted Ascendis a non-exclusive, worldwide license to use navepegritide, the active ingredient in the latter company’s recently approved achondroplasia drug Yuviwel, in exchange for 1…
    BioSpace · 7d agoApprovalPositive↗ source
  • Takeda and Protagonist Therapeutics receive FDA approval for rusfertide (Mimrylo) to treat polycythemia vera
    The FDA approved rusfertide, to be sold as Mimrylo, for the treatment of polycythemia vera, a rare blood cancer, marking a key win for Takeda and its partner Protagonist Therapeutics. The decision…
    BioSpace · 7d agoApprovalPositive↗ source
  • US biotech edge erodes as China overtakes in trials and research, raising security concerns
    The United States is losing its lead in biotech as China now conducts more clinical trials and publishes more natural‑science papers. China passed the U.S. six years ago in trial numbers and soon…
    BioSpace · 7d agoNegative↗ source