Pharming launches Joenja for APDS patients in Japan

Pharming has announced the commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients aged 4 years and older with activated PI3K delta syndrome (APDS). Following its inclusion on the Japan National Health Insurance drug price list, Japan has become the first global market where Joenja is commercially available for children aged 4 to 11.

Approved in March by Japan’s Ministry of Health, Labour and Welfare, Joenja is the first treatment specifically indicated for APDS. APDS is a rare primary immunodeficiency caused by genetic abnormalities that lead to an overactive PI3K-delta signaling pathway, resulting in symptoms such as recurrent infections, immune system disruption, and lymph node swelling.

Under an agreement with Pharming, OrphanPacific, Inc. serves as the Marketing Authorization Holder in Japan and is responsible for the supply and distribution of the medication. The approval was supported by positive results from global Phase III programs, including studies involving children aged 4 to 11.