Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
Pharming’s Joenja, an oral, selective PI3Kδ 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 U.S. in early April and will be available for shipment in mid-April.
Eveline Wu, division chief, Paediatric Rheumatology & associate professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, said: “The FDA approval of Joenja is an exciting moment for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that they will, for the first time, have access to an approved treatment, which has the potential to change the standard of care for the patient population suffering from APDS.”
Vicki Modell, co-founder of the Jeffrey Modell Foundation, an international, non-profit, organization dedicated to helping individuals and family members affected by primary immunodeficiency disorders, added: “The approval of Pharming’s Joenja is an important step toward making a difference in the lives of individuals living with APDS who experience severe, life-altering and progressive symptoms. The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.”
Sijmen de Vries, chief executive officer of Pharming, said: “This FDA approval of Joenja is an important milestone for people living with APDS who will now have access to the first approved treatment option specifically for this debilitating disease. Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS. We are grateful to the patients, caregivers, and physicians who participated in the clinical trials who have made today’s approval a reality.”
APDS is a rare primary immunodeficiency first characterized in 2013. It 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 upper and 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.
Pharming priority review voucher
With the approval of Joenja as a treatment for a rare pediatric disease, the FDA granted Pharming, a priority review voucher (PRV). The terms of Pharming’s 2019 exclusive license agreement with Novartis for leniolisib mean that Novartis has the right to purchase the PRV from Pharming, for a small minority share of the value of the PRV. Pharming will make milestone payments to Novartis and another party for the approval and first commercial sale for APDS totaling $10.5 million, and Pharming will make additional milestone payments to Novartis in an aggregate amount of up to $190 million upon the achievement of certain leniolisib sales milestones. The company also agreed to make tiered royalty payments to Novartis, calculated as low double-digit to high-teen double-digit percentage of net sales of leniolisib.
The Marketing Authorisation Application (MAA) for leniolisib with the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) is currently under review. Pharming expects that CHMP will issue its opinion on the MAA in the second half of 2023.