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168 stories matching this filter · ← front page

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  • Scribe Therapeutics’ early-stage IPO surges 43% as epigenetic cholesterol drug enters clinic
    Scribe Therapeutics filed for an IPO while its lead epigenetic therapy moves into early clinical testing, a rare combination in a market that prefers later-stage, de-risked offerings. The company…
    BioSpace · 4h agoPositive↗ source
  • Regeneron secures FDA approval for Pasatru, second U.S. treatment for rare bone disease FOP
    Regeneron received FDA approval for its antibody garetosmab, marketed as Pasatru, for treatment of fibrodysplasia ossificans progressiva (FOP) in adults. The approval makes Pasatru the second ther…
    BioSpace · 6h agoApprovalPositive↗ source
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Infectious Disease354
  • 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 · 8h agoApprovalPositive↗ source
  • Layoffs hit over 1,300 workers across seven biopharma M&A deals in 2026
    Workforce reductions this year have affected at least seven biopharma companies that recently completed mergers or acquisitions, eliminating a minimum of 1,359 jobs, according to BioSpace data. Th…
    BioSpace · 12h agoLayoffsNegative↗ source
  • Ultragenyx secures first gene therapy approval for GSDIa as Sanfilippo asset decision pending
    Ultragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa). The approval comes as the company awaits a secon…
    BioSpace · 7h agoApprovalPositive↗ source
  • Pediatric rare cancer research draws steady investment despite biotech market volatility
    The article notes that pediatric rare cancers continue to attract investment because they address the greatest unmet medical needs and allow clear measurement of patient impact. Even as the biotec…
    BioSpace · 15h agoPositive↗ 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 · 1d agoApprovalPositive↗ source
  • Takeda’s FDA-approved Orzeyful expands treatment to all narcolepsy symptoms, challenging existing sleep drugs
    The U.S. Food and Drug Administration approved Takeda's drug Orzeyful in August, marking the first therapy that addresses the full spectrum of narcolepsy symptoms, including cataplexy and excessive…
    BioSpace · 1d agoApprovalPositive↗ source
  • Trump nominates White House aide Heidi Overton as FDA commissioner
    President Donald Trump is reported to be selecting Deputy Director of the White House Domestic Policy Council Heidi Overton, a physician, to be the next FDA commissioner, pending Senate confirmatio…
    BioSpace · 1d agoRegulatoryNeutral↗ 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 · 1d agoLayoffsNegative↗ source
  • BioMarin to acquire Alesta Therapeutics for $275 million to expand bone disease pipeline
    BioMarin Pharmaceutical announced it will purchase Alesta Therapeutics for an upfront payment of $275 million. Alesta is developing an oral small-molecule drug, ALE1, aimed at treating hypophospha…
    Fierce Biotech · 2d agoDealPositive↗ source
  • LEO Pharma spends $435 million to acquire rare-disease skin drug dersimelagon
    LEO Pharma announced it will pay $435 million to buy the rights to dersimelagon, an oral MC1R agonist originally developed by Tanabe Pharma. The drug has completed Phase 3 trials for two rare phot…
    BioSpace · 2d agoDealPositive↗ source
  • Amylyx to seek FDA approval for avexitide after Phase 3 success in post-bariatric hypoglycemia
    Amylyx announced plans to file a new drug application for avexitide, a GLP-1 receptor antagonist, after a Phase 3 trial showed reduction in serious hypoglycemia in patients who had gastric bypass s…
    BioSpace · 2d agoRegulatoryPositive↗ 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 · 2d 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 · 2d agoDealNeutral↗ source
  • AI and data sharing essential for safer cell and gene therapies, says industry leader
    The author argues that keeping data secret in cell and gene therapy harms patients, and that a commitment to transparency and artificial intelligence is needed to make these treatments safer and mo…
    BioSpace · 2d agoPositive↗ source
  • Amylyx's avexitide halves hypoglycemia episodes in phase 3 trial for post‑bariatric patients
    Amylyx Pharmaceuticals reported that its GLP‑1 receptor antagonist avexitide significantly reduced dangerous blood‑sugar drops in a phase 3 study. The double‑blind Lucidity trial enrolled 78 indiv…
    Fierce Biotech · 3d agoPositive readoutPositive↗ source
  • AstraZeneca halts late-stage lung cancer trial of volrustomig after disappointing interim results
    AstraZeneca announced it is stopping a Phase 3 study of its bispecific antibody volrustomig in metastatic non-small cell lung cancer after an interim analysis showed the drug is unlikely to meet it…
    BioSpace · 3d agoNegative readoutNegative↗ source
  • AstraZeneca halts late-stage lung cancer trial of volrustomig after disappointing interim results
    AstraZeneca said it is ending its Phase 3 eVOLVE-Lung02 trial that evaluated the bispecific antibody volrustomig combined with chemotherapy as a first-line option for metastatic non-small cell lung…
    BioSpace · 3d agoNegative readoutNegative↗ 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 · 3d agoPositive readoutPositive↗ source
  • Leo Pharma signs $435 million deal with Tanabe for rare skin disease drug dersimelagon
    Leo Pharma announced a licensing agreement with Mitsubishi Tanabe Pharma for worldwide rights to dersimelagon, an oral melanocortin-1 receptor agonist targeting rare genetic skin disorders. The dr…
    Fierce Biotech · 3d agoDealPositive↗ source
  • Merck KGaA's US research site to cut 20 jobs amid broader R&D downsizing
    Merck KGaA's North American division, EMD Serono, announced it will lay off 20 employees at its Billerica, Massachusetts research site. The cuts are slated to take effect in the second half of Oct…
    Fierce Biotech · 3d agoLayoffsNegative↗ 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
  • EU regulator withdraws Amgen's Tavneos authorization over serious trial breaches
    European regulators have withdrawn the marketing authorization for Amgen's rare disease drug Tavneos, citing serious breaches in the pivotal clinical trial. The EMA said study staff from ChemoCent…
    BioSpace · 6d agoRegulatoryNegative↗ source
  • Two undisclosed gene-therapy deaths raise doubts about China's fast-track trial pathway
    Two recent deaths in China’s early‑stage gene‑therapy trials have drawn international attention. Both patients suffered severe immune reactions after receiving high‑dose viral vector treatments, an…
    Fierce Biotech · 6d agoRegulatoryNegative↗ source