FDA approval of Joenja as the first and only treatment indicated for activated phosphoinositide 3-kinase delta syndrome.- Pharming NV
Pharming Group N.V. announces that the FDA has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja, an oral, selective PI3K delta inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
The FDA evaluated the Joenja application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja is expected to launch in the US in early April and will be available for shipment in mid-April.
APDS is a rare primary immunodeficiency that was first characterized in 2013 and is currently estimated to affect 1 to 2 people per million. It is caused by genetic variants in either one of two identified genes, known as PIK3CD or PIK3R1, which are vital to the normal development and function of immune cells in the body. While people with APDS may suffer from a wide variety of symptoms, the most common are frequent and severe infections of the ears, sinuses, and upperand lower respiratory tracts. Infections usually begin in infancy. People with APDS are susceptible to swollen lymph nodes or an enlarged spleen (splenomegaly), as well as autoimmunity and inflammatory symptoms. People with APDS may also be at higher risk for cancers like lymphoma.
More than 200 patients have been identified around the world.
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