Leniolisib Becomes First FDA-Approved Treatment for Rare Immunodeficiency Disease

Author(s):

Phase 2/3 data show significant improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells in patients with activated phosphoinositide 3-kinase delta syndrome.

Leniolisib Becomes First FDA-Approved Treatment for Rare Immunodeficiency Disease

The US Food and Drug Administration (FDA) approved leniolisib (Joenja) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. According to an announcement from Pharming, the drug is anticipated to launch in early April.¹

The oral, selective PI3Kδ inhibitor is the first approved therapy for individuals living with APDS, a rare primary immunodeficiency disease affecting 1-2 people per million. The condition results from genetic variants in either PIK3CD or PIK3R1 genes.

"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,” Eveline Wu, MD, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, said in a statement.

Findings from the phase 2/3 data supported the agency’s decision. The multinational, triple-blind, placebo-controlled, clinical trial demonstrated significant improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells.

In the study of 31 patients with APDS, patients treated with a twice daily regimen of leniolisib 70mg exhibited significant progress compared with placebo, which fulfilled both endpoints.

"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,” Vicki Modell, co-founder, Jeffrey Modell Foundation stated.

References:

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS. News release. Pharming. Published March 24, 2023. Accessed March 24, 2023. https://prnmedia.prnewswire.com/news-releases/pharming-announces-us-fda-approval-of-joenja-r-leniolisib-as-the-first-and-only-treatment-indicated-for-apds-860863616.html