About Pharming Group N.V.
Pharming Group N.V. is a biopharmaceutical company specializing in the development and commercialization of protein replacement therapies and precision medicines for rare diseases. The company operates in the United States, Europe, and internationally.
Key Products
- RUCONEST – A recombinant C1 esterase inhibitor for treating acute hereditary angioedema (HAE) attacks in adults and adolescents.
- Joenja (leniolisib) – An oral small molecule PI3Kẟ inhibitor for activated phosphoinositide 3-kinase delta syndrome.
Pipeline Development
The company is advancing OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for HAE treatment.
Strategic Collaborations
- Development and license agreement with Novartis.
- Strategic partnership with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105.
Company Background
Founded in 1988, Pharming Group N.V. is headquartered in Leiden, the Netherlands.