Haegarda® (C1 esterase inhibitor subcutaneous [human]) gained US Food and Drug Administration (FDA)-approval in 2017, becoming the first subcutaneous C1 esterase inhibitor (C1-INH) approved for the treatment of hereditary angioedema (HAE). Haegarda is a human plasma-derived, purified, pasteurized, lyophilized concentrate prepared from large pools of human plasma.1 It is indicated for routine prophylaxis to prevent…
![Ruconest (C1 Esterase Inhibitor [Recombinant])](https://www.rarediseaseadvisor.com/wp-content/uploads/sites/25/2022/06/GettyImages-1227349947-544x306.jpg)
Ruconest® is a recombinant C1 esterase inhibitor indicated for the treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents.1 This is a prescription medication developed by Pharming Technologies B.V. that was approved by the European Medicines Agency (EMA) in 2010 and the US Food and Drug Administration (FDA) in 2014.1,2 Hereditary angioedema…
Kalbitor® is a plasma kallikrein inhibitor indicated for treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older.1 Mechanism of Action and Use of Kalbitor HAE occurs when a mutation in the gene that codes for C1-esterase inhibitor (C1-INH) protein results in deficient or absent C1-INH activity and low…
![Cinryze (C1-esterase Inhibitor [Human])](https://www.rarediseaseadvisor.com/wp-content/uploads/sites/25/2022/06/Cinryze-544x306.jpeg)
Cinryze®, manufactured by ViroPharma Biologics, part of Takeda, was approved by the US Food and Drug Administration (FDA) in October 2008 for routine prophylaxis in persons at least 6 years of age with acute attacks of hereditary angioedema (HAE).1 The European Medicines Agency (EMA) approved Cinryze in 2011, and in 2017, the FDA expanded its…
Pulmonary arterial hypertension (PAH) is described as a resting mean pulmonary arterial pressure (mPAP) of 25 mm Hg or higher, pulmonary capillary wedge pressure (PCWP) below 15 mm Hg, and pulmonary vascular resistance (PVR) above 3 Wood units in the absence of one of the common causes of pulmonary hypertension, such as left-sided heart disease,…
Diuretics are commonly prescribed to patients who have pulmonary arterial hypertension (PAH) with fluid retention.1 In PAH, vasoconstriction caused by endothelial and smooth muscle cell dysfunction, remodeling of the pulmonary arterial wall, and thrombosis results in right ventricular (RV) afterload.2 The increase in pulmonary vascular resistance leads to RV dilatation as well as RV diastolic…
Roctavian (valoctocogene roxaparvovec, AAV5-hFVIII-SQ), called ValRox until May 2020, is an investigational gene therapy under regulatory review for the treatment of severe hemophilia A, defined as a factor VIII level of 1 IU/dL or lower.1 Hemophilia A is a genetic disease caused by a deficiency of clotting factor VIII. It occurs much more frequently in…
Vasodilators have served as the foundation for pulmonary arterial hypertension (PAH) treatment since 1996. They target the vasculature systemically rather than the pulmonary vessels specifically.1 Vasodilators decrease systemic blood pressure and pulmonary arterial pressure and resistance by reversing vasoconstriction, improving systemic circulation and oxygenation, and decreasing cardiac load.2 Flolan (Epoprostenol) Flolan® (epoprostenol sodium) is an…
Orladeyo® (berotralstat) is an oral therapy indicated for the prevention of hereditary angioedema (HAE) attacks in children who are at least 12 years of age.1 Orladeyo is a registered trademark of BioCryst Pharmaceuticals, Inc and was first approved by the US Food and Drug Administration (FDA) in December 2020.2 This therapy was the first oral…
![Berinert C1-esterase Inhibitor [Human]](https://www.rarediseaseadvisor.com/wp-content/uploads/sites/25/2022/06/shutterstock_2164967999-544x306.jpg)
Berinert® is a human plasma-derived, lyophilized concentrate of C1-esterase inhibitor (C1-INH) indicated for the treatment of acute attacks of hereditary angioedema (HAE) affecting the abdomen, face, and larynx.1,2 HAE is a rare condition with an autosomal-dominant pattern of inheritance in which fluids accumulate outside blood vessels.3,4 The accumulated fluid blocks the flow of blood and…
