Pharming Group (NASDAQ:PHAR), a global biopharmaceutical company dedicated to transforming the lives of patients with rare diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion.
The committee recommends the marketing authorization of Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents aged 12 years and older.
The positive opinion marks a critical step toward providing the first targeted treatment for APDS patients in the European Union.
APDS is a rare and progressive primary immunodeficiency that causes significant immune dysregulation and increased susceptibility to severe, recurrent infections.
The CHMP’s recommendation is based on robust data from a randomized Phase II/III clinical trial involving 31 patients, which demonstrated statistically significant improvements in lymphoproliferation and immune function.
The filing was further supported by long-term, open-label extension data from 37 patients with a median exposure of three years, confirming a sustained safety and efficacy profile.
Following this recommendation, the European Commission is expected to issue a final decision on the marketing authorization in the second quarter of 2026.
This timeline aligns with Pharming’s broader global commercialization strategy, as Joenja has already secured regulatory approvals in the United States, the United Kingdom, and Japan.
Notably, the Japanese approval recently expanded the drug's reach to pediatric patients as young as four years old, a demographic Pharming is also pursuing in other major territories.