Pharming Group Projects 2025 Sales Surge
Pharming Group is forecasting a significant sales increase for 2025, driven by the expansion of its rare disease portfolio, including its drug Joenja. The company's optimistic projections, shared at the Oppenheimer Healthcare Conference, highlight the commercial potential in the rare disease market and the importance of manufacturing agility for CDMOs serving this sector.
Joenja, or leniolisib, is the first and only FDA-approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency. The agency granted it priority review, orphan drug, and rare pediatric disease designations before its approval on March 24, 2023, for patients 12 years and older. APDS, first identified in 2013, affects an estimated 1 to 2 people per million and is caused by genetic mutations that lead to immune system impairment. Pharming's preliminary unaudited revenues for 2025 were approximately $376 million, a 27% increase over 2024's $297.2 million. This performance surpassed the company's revised guidance of $365-$375 million. For 2026, the company forecasts revenue between $405 million and $425 million, representing 8% to 13% growth. The company is pursuing a label expansion for Joenja to include pediatric patients. A supplemental New Drug Application for children aged 4 to 11 is under priority review by the FDA with a decision expected by January 31, 2026. However, the company has excluded potential pediatric revenue from its 2026 guidance pending resolution of FDA questions on dosing. While Joenja's synthesis is outsourced to contract manufacturing organizations (CMOs), Pharming's other key product, Ruconest, utilizes a complex in-house manufacturing process. Ruconest is a recombinant C1 esterase inhibitor produced from the milk of transgenic rabbits, a method described as difficult to replicate. After initially planning a new downstream processing facility, Pharming renewed a long-term manufacturing partnership with Sanofi for Ruconest's production. The broader market for cell and gene therapy CDMOs is projected to grow significantly, expanding from $8.2 billion in 2025 to an estimated $75.32 billion by 2034. This growth is driven by the demand for treatments for rare diseases and genetic disorders. The rare disease small-batch CDMO market, in particular, is benefiting from regulatory incentives and the rise of precision medicine, with North America holding the dominant market share in 2024.
