Pharming Group has commercially launched Joenja (leniolisib) in Japan for adult and paediatric patients aged 4 years and older with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS), following its inclusion on Japan’s National Health Insurance (NHI) drug price list.

The launch makes Japan the first market where Joenja is commercially available for children between 4 and 11 years of age. The treatment received approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) in March 2026 as the first specifically approved treatment for APDS.

Under an agreement with Pharming, OrphanPacific serves as the Marketing Authorisation Holder for Joenja in Japan and is responsible for the product’s supply and distribution in collaboration with Pharming.

Leverne Marsh, Chief Commercial Officer of Pharming, said the Japanese launch represents an important milestone in the company’s global expansion and its efforts to provide treatment options to rare disease communities. She also acknowledged OrphanPacific’s role in bringing the treatment to eligible patients in Japan.

APDS is a rare primary immunodeficiency caused by genetic variants affecting the PIK3CD or PIK3R1 genes. The condition can result in immune dysfunction and is associated with recurrent respiratory infections, lymphoproliferation, autoimmunity and gastrointestinal complications. The disease affects an estimated one to two people per million worldwide.

Joenja is an oral small-molecule PI3K delta inhibitor designed to regulate activity in the PI3K delta pathway. Its approval is supported by results from a randomised, placebo-controlled Phase 3 clinical trial that demonstrated statistically significant improvements in its co-primary endpoints. Long-term data from an open-label extension have also supported the treatment’s safety and tolerability.

Joenja is approved for APDS in several markets, including the US, UK, European Union, Australia, Israel, Canada, South Korea and Saudi Arabia for patients aged 12 years and older, while Japan’s approval covers patients aged 4 years and older.

Leniolisib is also under regulatory review for paediatric APDS indications in several countries and is being evaluated in Phase II trials for other primary immunodeficiencies involving immune dysregulation.