Pharming Group N.V.

Pharming Group N.V. is a global biopharmaceutical company specializing in the development and commercialization of innovative protein replacement therapies and precision medicines for rare, debilitating, and life-threatening diseases. Its lead product, RUCONEST, is a recombinant C1 esterase inhibitor used to treat acute attacks in adult and adolescent patients with hereditary angioedema (HAE), demonstrating strong demand and double-digit revenue growth over a decade post-launch. Another key therapy, Joenja (leniolisib), is the only approved oral small molecule PI3Kδ inhibitor for activated phosphoinositide 3-kinase delta syndrome (APDS), with ongoing expansions into pediatric indications, geographic markets, and additional primary immunodeficiencies (PIDs) like those with immune dysregulation and common variable immunodeficiency (CVID). The company is advancing KL1333 for primary mitochondrial disease (PMD) in a registrational Phase II trial, alongside investigational therapies like OTL-105 for HAE. Through strategic collaborations with partners such as Novartis and Orchard Therapeutics, Pharming Group N.V. leverages specialized bioprocessing and a robust pipeline to address unmet needs in rare disease markets worldwide, headquartered in Leiden, Netherlands since its incorporation in 1988.