Tryvio-Aprocitentan
Tryvio is an oral tablet that lowers blood pressure in adults whose hypertension isn’t managed well despite using multiple medications. It blocks endothelin receptors to relax blood vessels, reducing risks of stroke and heart attack.
Duvyzat-Givinostat
Duvyzat is an oral histone deacetylase inhibitor prescribed for Duchenne muscular dystrophy in patients aged six and older. It slows muscle degeneration, reduces inflammation, and supports muscle strength by modulating gene regulation processes.
Vafseo-Vadadustat
Vafseo is an oral tablet that treats anemia in adults with chronic kidney disease on dialysis by boosting natural erythropoietin production through HIF‑prolyl hydroxylase inhibition. It minimizes the need for blood transfusions while carrying a caution for cardiovascular and clotting risks .
Wainua-Eplontersen
Wainua is a subcutaneous injection designed for adults managing nerve damage caused by hereditary transthyretin-mediated amyloidosis (ATTRv‑PN). It works via an antisense therapy that reduces the abnormal protein buildup damaging the nerves.
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.
