Approved
FDA Approves Attruby (Acoramidis) for ATTR-CM, Offering New Hope for Patients with Transthyretin Cardiac Amyloidosis
Attruby, Acoramidis, FDA Approval, ATTR-CM, Transthyretin Cardiac Amyloidosis, BridgeBio Pharma, Heart Disease Treatment
UCB’s Bimzelx Receives FDA Approval for Moderate-to-Severe Hidradenitis Suppurativa, Marking Fifth Indication
Bimzelx, Bimekizumab, Hidradenitis Suppurativa, FDA Approval, IL-17A and IL-17F Inhibitor, Chronic Inflammatory Skin Disease
FDA Panel Raises Safety Concerns Over AstraZeneca’s Anticoagulant Reversal Drug Andexxa
AstraZeneca, Andexxa, FDA panel, anticoagulant reversal drug, safety concerns, full approval
Pfizer’s Breakthrough Hemophilia Drug Hympavzi Wins EU Approval
Pfizer, Hympavzi, EU approval, hemophilia A, hemophilia B, marstacimab, once-weekly subcutaneous treatment, pre-filled auto-injector pen
Regenxbio Advances Duchenne Muscular Dystrophy Gene Therapy to Pivotal Studies, Potentially Challenging Sarepta’s Dominance
Regenxbio, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), Gene Therapy, RGX-202, Elevidys, Pivotal Studies, Biologics License Application (BLA), FDA Approval
FDA Approves Revuforj, a First-in-Class Menin Inhibitor, for Relapsed or Refractory Acute Leukemia with KMT2A Translocation
FDA approval, Revuforj, Menin inhibitor, Acute leukemia, KMT2A translocation, Relapsed or refractory, Syndax Pharmaceuticals
FDA Approves Danziten: A Breakthrough Nilotinib Treatment for Chronic Myeloid Leukemia Without Mealtime Restrictions
Danziten, Nilotinib, Chronic Myeloid Leukemia (CML), FDA Approval, Mealtime Restrictions, Azurity Pharmaceuticals
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder
FDA Denies Full Approval for Ocaliva in Primary Biliary Cholangitis, Accelerated Approval Stands
FDA, Ocaliva, Intercept Pharmaceuticals, primary biliary cholangitis, accelerated approval, rare liver disease
Syndax’s Revumenib Shows Promising Results in Acute Leukemia Trials, Paving Way for FDA Approval
Revumenib, Acute Leukemia, AUGMENT-101, SAVE Trial, BEAT AML, Syndax Pharmaceuticals, FDA Approval