Dutch biopharmaceutical company Pharming Group N.V. (PHAR, PHGUF,,PHARM.AS) announced Tuesday that Japan's Ministry of Health, Labour and Welfare or MHLW has approved Joenja (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, to treat activated PI3K delta syndrome or APDS in adult and pediatric patients aged 4 years and older.
With this approval, Joenja becomes the first treatment approved in Japan specifically for APDS and the first approved treatment anywhere globally, for children aged 4 to 11 with the disease.
Under a deal with Pharming, OrphanPacific, Inc. serves as the Marketing Authorization Holder for Joenja in Japan and is responsible for supply and distribution of the product in collaboration with Pharming.
The launch of Joenja is expected following agreement with the MHLW on its National Health Insurance drug price.
APDS is a rare primary immunodeficiency that was first characterized in 2013. It is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body.
Leniolisib, the PI3K inhibitor, was approved as the first and only targeted treatment of APDS in the U.S., U.K., Australia and Israel in adult and pediatric patients 12 years of age and older and in Japan for patients 4 years of age and older.
The approval in Japan was based on the data from Pharmings Phase III clinical program for leniolisib in APDS, including the global, randomized, placebo-controlled study in patients aged 12 years and older. In the trial, leniolisib met its co-primary endpoints by significantly reducing lymphadenopathy and significantly increasing the percentage of nave B cells out of total B cells.
The Japanese Pharmaceuticals and Medical Devices Agency or PMDA evaluated the application under the Priority Review pathway following the Orphan Drug Designation granted by the MHLW in May 2023.
Leverne Marsh, Chief Commercial Officer of Pharming, stated, "As the first treatment approved specifically for APDS in the country, it is the only targeted treatment option for patients and families affected by this rare and progressive disease. It also marks the first approval globally for children aged 4 to 11 with APDS. Together with OrphanPacific, we look forward to working through the next steps to help make Joenja available to patients in Japan as quickly as possible."
Leniolisib is currently under regulatory review for the treatment of APDS in the European Economic Area, Canada and several other countries. Leniolisib is also being evaluated in two Phase II clinical trials in primary immunodeficiencies (PIDs) with immune dysregulation.
The company noted that the safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS.
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