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78 stories matching this filter · ← front page

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  • Encoded Therapeutics raises $275 million in Series F round for Dravet syndrome program
    Encoded Therapeutics announced closing a Series F financing round that brought in $275 million. The round was led by GV with participation from an undisclosed healthcare investor. The new capital…
    Endpoints · 2d agoDealPositive↗ source
  • Pharvaris reports injectable-like efficacy from once-daily oral pill for rare genetic disease
    Pharvaris announced that its experimental oral formulation of deucrictibant achieved efficacy comparable to injectable versions in a once-daily pill. The drug is an extended-release version intend…
    Endpoints · 3d agoPositive readoutPositive↗ source
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Psychiatry & CNS882
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Endocrinology & Metabolism493
Infectious Disease456
  • Beam Therapeutics reports one-year data from early-stage AATD gene therapy trial
    Beam Therapeutics released new results from its early-stage gene-editing program targeting alpha-1 antitrypsin deficiency (AATD), a rare condition that can damage the lungs and liver. The data com…
    Endpoints · 4d agoPositive readoutPositive↗ source
  • 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 · 7d agoApprovalPositive↗ source
  • 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
  • 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 · 9d agoNegative readoutNegative↗ source
  • Medline issues correction for convenience kits with potentially non-sterile BD ChloraPrep applicators
    Medline has sent a correction notice after the FDA identified that some BD ChloraPrep applicators included in its convenience kits may not be sterile. The agency says the devices could cause tempo…
    fda medwatch · 10d agoCRLNeutral↗ source
  • 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 Takeda and Protagonist's rusfertide (Mimrylo) for polycythemia vera
    The U.S. Food and Drug Administration has granted approval to rusfertide, a therapy developed jointly by Takeda Pharmaceutical and Protagonist Therapeutics. The drug, to be marketed under the name…
    Endpoints · 14d agoApprovalPositive↗ source
  • FDA approves Roivant's oral dermatomyositis drug Lisraya (brepocitinib)
    The U.S. Food and Drug Administration has granted approval to Roivant's drug brepocitinib, which will be sold under the brand name Lisraya. The decision marks the first major regulatory clearance f…
    Endpoints · 15d 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 · 16d agoApprovalPositive↗ source
  • J&J’s Imaavy gains first-in-class label for warm autoimmune hemolytic anemia
    J&J announced that its monoclonal antibody Imaavy has received a label expansion in the United States. The expanded indication makes Imaavy the first therapy approved specifically for warm autoimm…
    Endpoints · 17d agoApprovalPositive↗ source
  • Option-to-acquire structures stay uncommon amid biotech's busy M&A season
    Biotech companies are experiencing one of the busiest deal-making years on record, with a flood of announced mergers and acquisitions. Two recent transactions have highlighted the option-to-acquir…
    Endpoints · 18d agoDealNeutral↗ source
  • FDA grants approval to Ultragenyx's Genglycos gene therapy for ultra-rare glycogen storage disorder
    Ultragenyx announced that the U.S. Food and Drug Administration has approved its gene therapy called Genglycos. The therapy targets an ultra-rare glycogen storage disorder that can lead to severe…
    Endpoints · 22d agoApprovalPositive↗ source
  • FDA approves Regeneron’s garetosmab for ultra-rare bone-forming disease
    The FDA has granted approval to Regeneron's antibody garetosmab, which targets activin A, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare disorder that causes…
    Endpoints · 23d agoApprovalPositive↗ 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
  • Leo Pharma to license Tanabe’s oral drug for rare light-sensitive skin disease, paying up to $435 million
    Leo Pharma announced a licensing agreement with Japan's Tanabe Pharma for an oral treatment targeting two genetic skin disorders that cause pain when exposed to sunlight. Under the deal, Leo Pharm…
    Endpoints · 25d agoDealNeutral↗ source
  • BioMarin signs new deal for early-stage bone disease asset
    BioMarin recently halted development of a rare disease drug that it had acquired in a $270 million transaction. Within a week, the company announced another deal to acquire an early‑stage asset aim…
    Endpoints · 24d agoDealNeutral↗ source
  • Argenx reports Phase 3 success for Vyvgart Hytrulo in autoimmune myositis, despite subgroup shortfall
    Argenx announced that its subcutaneous antibody Vyvgart Hytrulo achieved its primary endpoint in a Phase 3 trial for autoimmune myositis, a rare group of inflammatory muscle diseases. The trial de…
    Endpoints · 25d agoPositive readoutPositive↗ source
  • European regulators withdraw Amgen's Tavneos over questionable data
    European regulators announced they are pulling Amgen's rare disease drug Tavneos from the European market after reviewing data they deemed incorrect and misleading. The authorities said the data s…
    Endpoints · 29d agoRegulatoryNegative↗ source
  • FDA issues early alert on non-sterile BD ChloraPrep applicators in Medline convenience kits
    The FDA's Center for Devices and Radiological Health (CDRH) released an early alert about a potential high‑risk issue with certain Medline convenience kits that contain BD ChloraPrep applicators th…
    fda medwatch · 1mo agoRegulatoryNeutral↗ source
  • Epicrispr raises $90 million Series C after promising early data on epigenetic FSHD therapy
    Epicrispr Biotechnologies announced a $90 million Series C financing round to advance its research program. The company shared early data suggesting that an epigenetic approach could address facio…
    Endpoints · 1mo agoDealPositive↗ source
  • Swiss startup Vaderis raises $152 million to fund Phase 3 trial of rare bleeding disorder therapy
    Vaderis Therapeutics, a 13‑person Swiss biotech, announced a $152 million Series B financing round aimed at advancing its experimental therapy into a Phase 3 study. The company is testing a treatm…
    Endpoints · 1mo agoDealPositive↗ source
  • Jazz Pharmaceuticals to acquire Actio Biosciences for $820 million upfront, targeting ultra-rare epilepsy
    Jazz Pharmaceuticals announced it will purchase Actio Biosciences, a company developing an ultra‑rare epilepsy therapy. The transaction includes an upfront cash payment of $820 million, with the p…
    Endpoints · 1mo agoDealPositive↗ source