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FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia
News | December 27, 2025

FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia

The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia


Lunit SCOPE matches pathologist accuracy in HER2 scoring for advanced biliary tract cancer
Clinical Trials | December 25, 2025

Lunit SCOPE matches pathologist accuracy in HER2 scoring for advanced biliary tract cancer

Study published in laboratory investigation, the official journal of the United States and Canadian Academy of Pathology


FDA nod to Boehringer Ingelheim’s JASCAYD for progressive pulmonary fibrosis in adults
Drug Approval | December 24, 2025

FDA nod to Boehringer Ingelheim’s JASCAYD for progressive pulmonary fibrosis in adults

The study found that nerandomilast effectively slowed lung function decline in patients, with discontinuation rates similar to placebo


Neurocrine’s Valbenazine fails to show benefit in largest dyskinetic cerebral palsy trial
Clinical Trials | December 24, 2025

Neurocrine’s Valbenazine fails to show benefit in largest dyskinetic cerebral palsy trial

Adverse events were consistent with valbenazine’s established safety profile


Bayer wins Japan nod for finerenone in broad heart failure population
Drug Approval | December 23, 2025

Bayer wins Japan nod for finerenone in broad heart failure population

Finerenone is the first therapy targeting the mineralocorticoid receptor pathway to demonstrate cardiovascular benefits in heart failure patients


FDA grants breakthrough therapy designation to Enhertu for high-risk early breast cancer
Drug Approval | December 23, 2025

FDA grants breakthrough therapy designation to Enhertu for high-risk early breast cancer

This Breakthrough Therapy Designation highlights the impressive clinical benefit of Enhertu over the current standard of care


FDA go-ahead to Roche’s one-minute follicular lymphoma therapy
Drug Approval | December 23, 2025

FDA go-ahead to Roche’s one-minute follicular lymphoma therapy

Full approval will depend on verification of clinical benefit in a confirmatory trial


Ipsen’s FALKON trial falls short in rare FOP study, offers key insights
Clinical Trials | December 22, 2025

Ipsen’s FALKON trial falls short in rare FOP study, offers key insights

FOP is a rare genetic disorder caused by mutations in the ALK2 kinase