The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on Pharming Group’s Joenja (leniolisib) for activated PI3K‑delta syndrome (APDS) on March 26, 2026. The opinion clears the way for a European Commission decision, expected in the second quarter of 2026, and would make Joenja the first approved treatment for APDS in the European Union.
Joenja is an oral, selective PI3Kδ inhibitor that targets the underlying immune dysregulation in APDS, a rare primary immunodeficiency that causes recurrent infections, lymphoproliferation and autoimmunity. The drug has already received regulatory approval in the United States (March 2023) and the United Kingdom, and was approved in Japan on March 24, 2026, covering patients aged four years and older. The CHMP opinion is based on a randomized Phase II/III trial that enrolled 31 patients, with long‑term data from 37 patients.
The regulatory milestone comes at a time when Pharming’s commercial performance is accelerating. In 2025 the company generated US$376.1 million in revenue, a 27 % year‑over‑year increase driven by sales of RUCONEST® and Joenja®. Operating profit rose to US$25.8 million, a turnaround from a loss in 2024. For the fourth quarter of 2025, revenue reached US$106.5 million, up 15 % YoY, and the company has guided 2026 revenue to US$405–425 million, reflecting confidence in the expanded EU market.
CEO Fabrice Chouraqui said, “For patients living with APDS, there remains a significant unmet medical need. By targeting the underlying cause of the disease, Joenja could mark a step‑change in APDS care in Europe. Today's positive CHMP opinion, together with the approval in Japan earlier this week, reflects the strength of the clinical data and the dedication of the patients, families, and physicians who participated in the clinical studies. We look forward to the European Commission's final decision and to working with relevant authorities across Europe to support patient access.”
The potential EU approval would open a new revenue stream for Pharming, expanding Joenja’s addressable patient base beyond the United States and strengthening the company’s position as a rare‑disease specialist. The rare‑disease market is characterized by limited treatment options and high unmet need, so a first‑in‑EU approval could drive significant growth and support the company’s broader pipeline strategy.
With the CHMP opinion in place, Pharming is positioned to secure the European Commission’s decision in the coming months, potentially accelerating the drug’s commercial launch in the EU and reinforcing the company’s trajectory of robust financial performance and strategic expansion.
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