Pharming Group secures first-ever APDS treatment approval in Japan for patients 4+

Pharming Group (NASDAQ:PHAR) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted marketing approval for Joenja (leniolisib) as the first targeted treatment for activated phosphoinositide 3-kinase delta syndrome (APDS).

The approval covers both adult and pediatric patients aged four years and older, representing a historic "first" for the global medical community as the earliest age-authorized use of the drug worldwide.

The MHLW’s decision was underpinned by positive Phase III clinical data demonstrating significant improvements in two hallmark indicators of APDS: the reduction of lymphadenopathy (swollen lymph nodes) and an increase in naïve B cells, which are essential for a healthy immune response.

The trial results showed that leniolisib was generally well-tolerated, with most adverse reactions limited to mild cases of headache, sinusitis, and atopic dermatitis.

In Japan, Pharming has partnered with OrphanPacific, which will serve as the Marketing Authorization Holder and manage the local supply and distribution.

The commercial launch is expected to commence immediately following the determination of the drug's price under Japan’s National Health Insurance (NHI) system.

This approval is particularly significant for the 4–11 age demographic; while Joenja is already marketed in the U.S. and U.K. for patients 12 and older, Japan is now the first territory to provide access to younger children who previously relied solely on supportive care.