• Joenja® (leniolisib) is first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients aged 12 years and older
• First European launch expected in Germany in Q3 2026
• Approval adds to Joenja® approvals in the United States, United Kingdom, Japan, Australia and Israel, supporting Pharming’s global expansion strategy

Pharming Group (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR)  announced that the European Commission (EC) has granted marketing authorization for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older. Joenja is the first and only approved treatment for APDS in the European Union (EU). The first European launch is expected in Germany in Q3 2026, with additional launches anticipated pending completion of national reimbursement negotiations.

Leverne Marsh, Chief Commercial Officer of Pharming, commented:
“APDS is a progressive disease that can have a profound, lifelong impact on those living with the condition and their families, significantly affecting quality of life and underscoring the need for targeted treatment options. Today’s approval marks an important milestone for the European APDS community, making Joenja the first therapy in Europe specifically indicated to treat this disease.

This milestone reinforces our commitment to tackling rare disease challenges where new options are urgently needed. Joenja has the potential to meaningfully change how this complex condition is managed. We are proud to work with healthcare providers, patient communities and reimbursement authorities to support timely availability for eligible individuals across Europe.”

The EC approval is based on results from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, evaluating leniolisib in 31 patients with APDS aged 12 years and older. The study demonstrated statistically significant improvements in markers of immune dysregulation and immunodeficiency. The application also included long-term, open-label extension data from 37 patients who received leniolisib for a median of three years.

Prof. Ulrich Baumann, MD, Managing Senior Physician, Department of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Germany commented:

“The available clinical data on leniolisib consistently demonstrated a significant improvement in key parameters of immune dysregulation in patients with APDS. Of particular note is its targeted approach, which directly targets the underlying pathophysiology. Against the background of previously limited therapeutic options, this approval represents an important advancement in the management of this patient population.”

The approval is valid across all 27 EU Member States, as well as Norway, Iceland and Liechtenstein. Joenja is currently marketed in the United States and the United Kingdom for patients aged 12 years and older with APDS.