Tavneos-Avacopan
ANCA-associated vasculitis treatment has depended on prolonged high-dose glucocorticoids, whose toxicity accumulates over a patient’s lifetime. Tavneos (avacopan) was developed to reduce that dependence. Avacopan is an oral C5a receptor antagonist that blocks the complement signal priming and recruiting neutrophils to damage blood vessels. It is approved as an adjunct to standard therapy, including glucocorticoids, […]
Livmarli-Maralixibat
Children with Alagille syndrome can experience itching severe enough to disrupt sleep and development, driven by bile acids the liver cannot clear. Livmarli (maralixibat) was the first therapy approved for it. By blocking bile acid reabsorption in the distal intestine, it causes bile acids to be excreted rather than recirculated, lowering the levels that drive […]
Qulipta-Atogepant
Qulipta (atogepant) belongs to the oral generation of CGRP therapies, which followed the injectable antibodies that first moved migraine prevention beyond repurposed drugs. As a CGRP receptor antagonist, it blocks the peptide signaling involved in triggering attacks, without the vasoconstriction that limits older triptans. The once-daily tablet is approved for the preventive treatment of migraine […]
Exkivity-Mobocertinib
EGFR exon 20 insertion lung cancer briefly had two targeted options approved within months of each other. Exkivity (mobocertinib) was the oral one, and it did not last. Designed to bind the altered EGFR conformation these insertions produce, the once-daily oral inhibitor held accelerated approval for locally advanced or metastatic non-small cell lung cancer with […]
Korsuva-Difelikefalin
Uremic pruritus affects a large share of hemodialysis patients and had no approved treatment, leaving clinicians to rely on antihistamines and off-label agents. Korsuva (difelikefalin) filled that gap. Difelikefalin is the first selective kappa opioid receptor agonist approved, acting on peripheral nerves and immune cells without meaningfully entering the brain. It is given intravenously at […]
Welireg-Belzutifan
Welireg (belzutifan) was the first HIF-2 alpha inhibitor approved, targeting the pathway unleashed when von Hippel-Lindau function is lost and tumors begin forming across multiple organs. By blocking HIF-2 alpha, the transcription factor that accumulates when VHL function is lost, Welireg interrupts the signal driving tumor formation. The once-daily tablet treats adults with von Hippel-Lindau […]
Bylvay-Odevixibat
Progressive familial intrahepatic cholestasis produces relentless itching from accumulated bile acids, and surgical biliary diversion was long one of the few options. Bylvay (odevixibat) provided a pharmacological alternative. Odevixibat blocks bile acid reabsorption in the distal intestine, lowering the serum levels that drive severe itch. The once-daily capsule, with a pellet formulation for young children, […]
Rezurock-Belumosudil
Chronic graft-versus-host disease becomes progressively fibrotic, and patients who fail multiple lines of immunosuppression have historically had little to fall back on. Rezurock (belumosudil) offered a different mechanism. As a first-in-class ROCK2 inhibitor, Rezurock rebalances the immune response toward regulatory T cells while directly reducing fibrosis. The once-daily tablet treats chronic graft-versus-host disease in patients […]
Fexinidazole-Fexinidazole
Human African trypanosomiasis was historically treated with regimens requiring hospitalization, lumbar puncture-based staging, and arsenic-derived drugs with lethal side effects. Fexinidazole changed the treatment model entirely. Unlike the arsenic-derived and injectable regimens it displaced, fexinidazole is a nitroimidazole taken once daily for 10 days, activated inside the parasite to damage its DNA and proteins. It […]
Kerendia-Finerenone
Diabetic kidney disease progresses despite blood pressure and glucose control, and steroidal mineralocorticoid antagonists carry hyperkalemia and hormonal side effects that limit their use. Kerendia (finerenone) was designed around those constraints. Finerenone is the first nonsteroidal selective mineralocorticoid receptor antagonist, targeting the inflammation and fibrosis that drive progression rather than acting mainly on blood pressure. […]
