Fazirsiran

Arrowhead Pharmaceuticals Inc. is developing Plozasiran, an RNAi therapeutic for Familial chylomicronemia syndrome (FCS), showing significant triglyceride reduction. Submitted NDA on Nov.18, 2024, with FDA review decision by Jan.18, 2025. Also exploring treatments for Severe Hypertriglyceridemia and Dyslipidemia.

Takeda's late-stage pipeline includes six programs with peak revenue potential of $10B - $20B, with Phase 3 data readouts expected in 2025 for Oveporexton, Zasocitinib, and Rusfertide. Regulatory filings for these drugs are planned for FY2025-2026, with additional filings anticipated through FY2029.

Alpha-1 antitrypsin deficiency (AATD) affects 1 in 3,500, causing liver/lung damage. Current treatment, augmentation therapy, doesn't reverse damage. Multiple companies develop candidates to prevent/reverse damage, with RNAi, RNA-editing, and base editing therapies showing promise. Vertex's AATD candidates failed, while Arrowhead, Sanofi, Beam Therapeutics, Wave Life Sciences, and Korro Bio advance their treatments. Augmentation therapy may become obsolete within 5-10 years.