Pharming Group N.V. announces the first commercial shipments of Joenja® to patients in the United States. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the U.S. for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency, in adult and pediatric patients 12 years of age and older.
Under the terms of Pharming's 2019 exclusive license agreement with Novartis for leniolisib, the corresponding first commercial sale of Joenja® triggers a $10 million milestone payment by Pharming to Novartis.
Stephen Toor, Chief Commercial Officer of Pharming, commented
"We are pleased to announce that the first Joenja® shipments to patients, with payor reimbursement, were delivered approximately two weeks following FDA approval, achieving an important milestone for patients suffering with APDS. We look forward to making Joenja® widely available across the U.S. as the first and only approved treatment for patients with APDS."
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS 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. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 A definitive diagnos
is can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
About Pharming Group N.V.
Pharming Group N.V. is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.