Joenja is indicated for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), which is caused by gain of function (GOF) mutation in the PIK3CD gene that encodes for p110δ catalytic subunit of PI3Kδ

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APDS is a rare type of immunodeficiency. (Credit: James Yarema on Unsplash)

Dutch biopharmaceutical company Pharming has received the US Food and Drug Administration (FDA) approval for Joenja (leniolisib) to treat a rare type of primary immunodeficiency.

Joenja is an oral, selective PI3Kδ inhibitor, indicated for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 years and above.

APDS is caused by a gain of function (GOF) mutation in the PIK3CD gene that encodes for p110δ catalytic subunit of PI3Kδ (APDS1).

PIK3CD, also called PIK3R1, plays an important role in the normal development and function of immune cells in the body.

According to the company, people with APDS may present various symptoms, with the most common being frequent and severe infections of the ears, sinuses, and upper and lower respiratory tracts.

The patients are prone to swollen lymph nodes or an enlarged spleen, along with autoimmunity and inflammatory symptoms, and may also be at elevated risk for developing cancers.

Joenja is the first and only treatment approved in the US for APDS and will be rolled out in the US, in early April, said the company.

Pharming chief executive officer Sijmen de Vries 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.

“I would also like to thank the Pharming and the Novartis teams who have supported the development of Joenja and can, therefore, be justifiably proud of this FDA approval.”

The US health regulator evaluated Pharming’s New Drug Application (NDA) for Joenja under priority review and has granted the approval based on results from a Phase 2/3 clinical trial.

The Phase 2/3 study evaluated the efficacy and safety of the drug in 31 patients diagnosed with APDS, aged 12 years and above.

In the study, Joenja showed significant improvement in lymphoproliferation, including a reduction in the size of lymph nodes and an increase in naïve B cells compared to placebo, which is the study’s co-primary endpoint.

Pharming has also submitted data from a long-term, open-label extension clinical trial in which 38 patients received Joenja for a median of two years, as part of the regulatory application.

Furthermore, the company’s Marketing Authorisation Application (MAA) for leniolisib is currently under review by the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP), with an opinion expected in the second half of 2023.