Biologically Active Substance
Novartis Takes Another Shot at Alpha-Synuclein With $2.2B Arrowhead CNS Deal
Novartis; Arrowhead Pharmaceuticals; alpha-synuclein; Parkinson’s disease; RNA interference (RNAi); gene silencing; ARO-SNCA; TRiM platform; neurodegeneration; biotech partnerships
Sanofi’s Wayrilz Receives FDA Approval as First BTK Inhibitor for Immune Thrombocytopenia
Wayrilz; rilzabrutinib; FDA approval; immune thrombocytopenia; ITP; BTK inhibitor; Sanofi; LUNA 3 trial
Servier Pays $210M Upfront for Ex-US Rights to Ideaya’s PKC Inhibitor Darovasertib
Servier; Ideaya Biosciences; darovasertib; protein kinase C inhibitor; PKC inhibitor; uveal melanoma; oncology licensing deal; clinical trials; milestone payments; royalties
Ionis Posts Strong Results for Tryngolza in Severe Blood Fat Disease
Ionis Pharmaceuticals; Tryngolza; olezarsen; hypertriglyceridemia; triglycerides; Phase III Essence study; FDA approval; familial chylomicronemia syndrome; cardiovascular risk reduction; drug launch
Recent Advances for Confident Cell Line Transfer
cell line development; cell transfer; clone stability; electroporation; transfection; quality assurance; GMP compliance; cell therapy
AbbVie’s Rinvoq Achieves Strong Phase 3 Results in Alopecia Areata, Clearing Key Efficacy Milestone
AbbVie; Rinvoq; Phase 3 trial; alopecia areata; hair regrowth; upadacitinib; SALT score; JAK inhibitor
FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
FDA rejection; PTC Therapeutics; vatiquinone; Friedreich’s ataxia; complete response letter; drug efficacy; additional clinical studies
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug
Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program
FDA Grants Wegovy 3rd Indication: First GLP-1 Approved for MASH with Moderate-to-Advanced Liver Scarring
Wegovy; FDA approval; MASH; semaglutide; GLP-1 receptor agonist; liver fibrosis; Novo Nordisk; ESSENCE trial; noncirrhotic; accelerated approval