Truseltiq™ (infigratinib), a kinase inhibitor, is indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as determined by a test approved by the US Food and Drug Administration (FDA).1 Cholangiocarcinomas (CCAs) are cancers that develop in…
Tibsovo® (ivosidenib) is a first-in-class targeted isocitrate dehydrogenase (IDH) 1 inhibitor indicated for adult patients carrying a susceptible IDH1 mutation as detected by a US Food and Drug Administration (FDA)-approved test who have been previously treated for advanced or metastatic cholangiocarcinoma (CCA).1 Tibsovo was approved by the FDA for use in patients with CCA in…
Hemgenix® (etranacogene dezaparvovec-drlb) is a gene therapy commercialized by CSL Behring that is indicated for the treatment of adult patients with hemophilia B who are undergoing factor IX (FIX) prophylaxis therapy, have a history of life-threatening hemorrhage, or experience repeated, serious, spontaneous bleeding events.1 Hemophilia B is an inherited disease derived from mutations in the…
Lovo-cel (also known as bb1111 or LentiGlobin™ for sickle cell disease [SCD]) is an investigational gene therapy in development by Bluebird Bio for patients with SCD. This experimental treatment is envisioned as a one-time therapy for patients with SCD.1 Sickle cell disease is a debilitating genetic disease caused by a single point mutation in the…
Ultomiris® (ravulizumab) is a US Food and Drug Administration (FDA)-approved humanized monoclonal antibody against the complement protein C5 that was developed by Alexion Pharmaceuticals. It is used to treat children (at least 1 month of age and 5 kg body weight) and adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare fatal disease characterized by intravascular…
Empaveli® is a drug indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH),1 a rare disease of the blood caused by the clonal expansion of mutated hematopoietic stem cells (HSCs).2 Stress triggered by infection or exertion can lead to complement activation and the complement-mediated lysis of red blood cells and platelets. Hemolysis and…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease caused by somatic mutations in the PIGA (phosphatidylinositol glycan A) gene in hematopoietic stem cells.1 The mutations result in a deficiency of glycosylphosphatidylinositol-anchored proteins, which render red blood cells susceptible to hemolysis. Patients who have PNH typically present with hemolytic anemia, bone marrow dysfunction, and thrombosis.1…
Soliris® was developed by Alexion Pharmaceuticals and was the first drug to be approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) by the US Food and Drug Administration (FDA), in 2007.1,2 Soliris is also approved for treatment of PNH in the European Union and Japan.2 Indications Soliris is indicated for the reduction of hemolysis…
Immune thrombocytopenia (ITP) is an autoimmune disease that leads to a low platelet count and an increased risk of bleeding. The first-line treatment of ITP aims to reduce bleeding and increase the number of platelets, which is often achieved with the administration of corticosteroids. Second- and third-line treatments include thrombopoietin receptor agonists (TPO-RAs), Rituxan® (rituximab),…
Promacta® (eltrombopag olamine) is an oral small molecule thrombopoietin receptor agonist (TPO-RA). It increases platelet synthesis by interacting with the TPO receptor’s transmembrane domain, also called cMpl.1 Promacta was approved by the FDA in 2008 for use in adult patients with immune thrombocytopenia. In 2015, the FDA approved Promacta for pediatric patients aged 1 year…
