Pharming Group: Joenja® Treatment Approved in Japan, a Historic First
Pharming Group announces the approval by the Japanese Ministry of Health, Labour and Welfare of Joenja® (leniolisib), a selective inhibitor of phosphoinositide 3-kinase delta, for the treatment of APDS in patients aged 4 years and older. This approval marks a first in Japan and is the first specific targeted treatment for this rare primary immunodeficiency approved in the country.
A Milestone Approval for APDS Treatment
Joenja becomes the first approved treatment in Japan specifically for APDS and the first globally approved treatment for children aged 4 to 11 years with this condition. The approval encompasses both adult and pediatric patients aged 4 years and older. The product is already approved and marketed in the United States and the United Kingdom for patients aged 12 years and older. The Japanese Pharmaceutical Agency (PMDA) reviewed the application through a priority assessment procedure following the orphan drug designation granted in May 2023.
Collaboration and Market Authorization
OrphanPacific, Inc. serves as the marketing authorization holder for Joenja in Japan and collaborates with Pharming for the supply and distribution of the product. The launch of Joenja is anticipated following an agreement with the MHLW on the pricing of the drug under the national health insurance. The approval is based on data from Pharming's Phase III clinical trial program, including a randomized placebo-controlled study in patients aged 12 years and older, an open Japanese study, as well as positive data from a multinational open Phase III study in children aged 4 to 11 years.
