Voydeya-Danicopan
Voydeya is an oral complement factor D inhibitor added to standard therapy in adults with paroxysmal nocturnal hemoglobinuria. It helps reduce ongoing hemolysis outside blood vessels, improving anemia.
Fabhalta-Iptacopan
Fabhalta is an oral capsule that helps treat rare blood and kidney diseases by inhibiting complement factor B. It’s approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), and to reduce proteinuria in complement 3 glomerulopathy (C3G) and IgA nephropathy.
Filsuvez-Birch Triterpenes
Filsuvez is a topical gel used on open wounds in patients with dystrophic or junctional epidermolysis bullosa (EB). It’s the first treatment that supports skin healing in this condition by promoting wound closure.
Zilbrysq-Zilucoplan
Zilbrysq is a once-daily self-administered injection for adults with generalized myasthenia gravis who test positive for anti-AChR antibodies. It blocks the complement C5 protein to reduce muscle weakness and improve strength.
Agamree-Vamorolone
Agamree is an oral suspension for Duchenne muscular dystrophy in children aged 2 and older. It’s a steroid-type medicine that helps protect muscles and slow disease progression.
Fruzaqla-Fruquintinib
Fruzaqla is a once-daily oral treatment for metastatic colorectal cancer in adults who have already tried other therapies. It inhibits VEGF receptors to slow tumor growth and improve survival.
Defencath-Taurolidine And Heparin
Defencath is a catheter lock solution used in adult dialysis patients with central venous catheters. It helps prevent bloodstream infections by combining antimicrobial and anti-clotting effects.
Augtyro-Repotrectinib
Augtyro is an oral treatment for ROS1-positive non-small cell lung cancer and NTRK fusion–positive tumors. It blocks key cancer-driving proteins to help slow tumor growth, especially in patients who’ve tried other therapies.
Truqap-CapIVasertib
Truqap is used with fulvestrant to treat certain advanced breast cancers with specific genetic mutations. It works by blocking the AKT pathway, which helps control tumor cell survival and growth.
Ogsiveo-Nirogacestat
Ogsiveo is an oral therapy for adults with desmoid tumors that are actively progressing. It targets a protein involved in tumor formation, helping reduce tumor size and manage symptoms.
