PHAR

Pharming Group N.V. is a biopharmaceutical company dedicated to the creation and commercialization of protein replacement therapies and precision medicines. The firm concentrates its efforts on addressing rare diseases and critical unmet medical needs across the United States, Europe, and other international markets. Its primary commercialized product is Ruconest, a recombinant human C1 esterase inhibitor utilized for the acute treatment of hereditary angioedema. Pharming's development pipeline also includes rhC1INH, which is being investigated for conditions such as pre-eclampsia, acute kidney injury, and COVID-19. Additionally, the company is developing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor designed for patients with activated PI3K delta syndrome, as well as an alpha-glucosidase therapy targeting Pompe and Fabry diseases. The company has established significant collaborations, including a development and licensing agreement with Novartis. It also maintains a strategic partnership with Orchard Therapeutics plc for the research, development, manufacturing, and commercialization of OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy intended for hereditary angioedema. Pharming Group N.V. is headquartered in Leiden, the Netherlands.