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Argenx is a Dutch biopharmaceutical company focused on developing antibody-based therapies for rare autoimmune diseases. The company's lead product, Vyvgart (efgartigimod), was approved by the FDA in December 2021 for the treatment of generalized myasthenia gravis (gMG). In 2022, Argenx also received FDA approval for Vyvgart Hytrulo, a subcutaneous formulation of Vyvgart, offering a more convenient option compared with Vyvgart's intravenous administration. In 2024, the FDA approved Vyvgart Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy, a rare immune-mediated neuromuscular disorder of the peripheral nervous system. Argenx is focused on innovation and developing its pipeline for treatments such as primary immune thrombocytopenia, thyroid eye disease, and Sjogren's Disease.
Alnylam Pharmaceuticals is a leader in the study of RNA interference (RNAi) therapeutics. RNAi is a naturally occurring biological pathway within cells for sequence-specific silencing and regulation of gene expression. Alnylam has five drugs on the market: Onpattro and Amvuttra for hATTR amyloidosis, Givlaari for acute hepatic porphyria, Oxlumo for primary hyperoxaluria type 1, and Leqvio for hypercholesterolemia. It also has several clinical programs across various therapeutic areas: genetic medicines; cardio-metabolic diseases; hepatic infectious diseases, and CNS/ocular diseases. Upfront fees from research partnerships have boosted Alnylam's cash levels, and the company stands to see additional milestones and royalties from drugs commercialized under collaboration partnerships.