U.S. Food and Drug Administration– tag –
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Patisiran and Inotersen Halt Polyneuropathy Progression in hATTR Amyloidosis; Next-Gen siRNA Vutrisiran Also FDA Approved
oneAMYLOIDOSISvoice USA Overview Patisiran (Onpattro) and inotersen (Tegsedi), TTR mRNA silencing therapies for hereditary ATTR amyloidosis (hATTR), have demonstrated the ability to halt the progression of polyneuropathy and even show im... -
Insilico Medicine’s AI Drug Discovery Accelerates: Lenticelib, First AI-Designed Target and Structure, Achieves IND in 18 Months and Enters Mid-Stage Clinical Trials
Forever.ai (Appears to be a scientific review/journal) Unknown Overview AI drug discovery is dramatically reducing early-stage discovery timelines from years to mere weeks or months, encompassing target selection, hit identification, lea... -
Exponential Progression of Targeted Protein Degradation: First FDA-Approved PROTAC and Molecular Glues Unlock “Undruggable” Targets
MarinBio (Appears to be a scientific review/journal, affiliated with MarinBio, hence using their domain) USA Overview The field of targeted protein degradation (TPD) is rapidly evolving, enabling the degradation of previously "undruggabl... -
Arvinas/Pfizer’s PROTAC Drug Veppanu Receives Historic FDA Approval for ESR1-Mutant Advanced Breast Cancer, Demonstrating 43% PFS Risk Reduction
Cancer Discovery - AACR Journals USA Overview Arvinas and Pfizer's oral PROTAC drug, Veppanu, has received FDA approval for advanced breast cancer with ESR1 mutations, marking the first clinical validation of targeted protein degradation... -
Ionis Enters Global Licensing Deal with Recordati for Alexander Disease ASO Zilganersen: Secures $30M Upfront Payment to Accelerate International Expansion
Rare Disease Report USA Overview Ionis Pharmaceuticals has entered a global licensing agreement with Recordati for zilganersen, an antisense oligonucleotide (ASO) for Alexander disease, covering territories outside the U.S. Ionis will re... -
Helus Pharma™’s Breakthrough-Designated Depression Drug HLP003 Completes 86% Enrollment in Phase 3 Trial, Topline Data Expected Q4 2026
PR Newswire Unknown Overview Helus Pharma™’s APPROACH Phase 3 trial for HLP003, an investigational adjunct therapy for major depressive disorder (MDD), has successfully completed over 86% of patient enrollment. HLP003 has received Breakt... -
Kowa’s Selective PPARα Modulator Pemafibrate Receives FDA Breakthrough Therapy Designation for Primary Biliary Cholangitis Treatment, Demonstrating Significant ALP Improvement in Phase II Data
Morningstar Japan Overview Japan's Kowa Company has announced that its selective PPARα modulator "K-808" (pemafibrate) has been granted Breakthrough Therapy designation by the U.S. FDA for the treatment of primary biliary cholangitis (PB... -
Ionis’ ASO Therapy Zilganersen Awaits FDA Approval for Alexander Disease: Achieves Primary Endpoints and Granted Priority Review
BioXconomy USA Overview Ionis Pharmaceuticals' antisense oligonucleotide (ASO), zilganersen, is awaiting FDA approval for the ultra-rare neurological disorder, Alexander disease. The pivotal Phase 1-3 trial (NCT04849741) met its primary ... -
FDA Approves Expanded Indication for Ionis’ ASO Tryngolza in Severe Hypertriglyceridemia, Achieving <500 mg/dL TG Levels in 86% of Patients
Fierce Pharma USA Overview The FDA has approved an expanded indication for Ionis Pharmaceuticals’ antisense oligonucleotide (ASO) Tryngolza (olezarsen) for severe hypertriglyceridemia. Clinical trials demonstrated remarkable efficacy, wi... -
Otsuka’s ADHD Therapy Poised for FDA Approval Following Successful Phase 3b Clinical Trial
Fierce Biotech USA Overview Otsuka Pharmaceutical announced positive outcomes from its Phase 3b clinical trial for an Attention-Deficit/Hyperactivity Disorder (ADHD) treatment, marking a crucial step ahead of its U.S. Food and Drug Admin...
