The treatment is approved in the US for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in both adults and paediatric patients aged 12 years and above. Credit: florentiabuckingham from Pixabay.

Pharming Group has conducted the first commercial shipments of oral selective PI3Kδ inhibitor Joenja (leniolisib) to patients diagnosed with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in the US.

Joenja marks the first and only treatment approved in the country for the rare and progressive primary immunodeficiency in both adults and paediatric patients aged 12 years and above.

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The latest shipment is based on an exclusive licence agreement signed in 2019 between Pharming and Novartis. Under its terms, Pharming has paid a $10m milestone payment to Novartis for the first commercial sale of Joenja.

In March 2023, Joenja secured approval from the US Food and Drug Administration (FDA) to treat the targeted patients.

Pharming chief commercial officer Stephen Toor said: “We are pleased to announce that the first Joenja shipments to patients, with payer reimbursement, were delivered approximately two weeks following FDA approval, achieving an important milestone for patients suffering with APDS.

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“We look forward to making Joenja widely available across the US as the first and only approved treatment for patients with APDS.”

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APDS is a rare primary immunodeficiency caused by variants in either one of two identified genes, PIK3CD or PIK3R1. Both genes are important for the body’s development and for the functioning of immune cells.

Variants of these genes cause hyperactivity of the PI3Kδ pathway, preventing immune cells from maturing and functioning properly and leading to immunodeficiency and dysregulation.