The U.S. Food and Drug Administration (FDA) has approved leniolisib (Joenja) to treat persons 12 years age an older with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
APDS is a rare primary immunodeficiency caused by genetic variants in either PIK3CD or PIK3R1 genes, both of which are vital to the normal development and function of immune cells in the body. Symptoms vary greatly in this rare disease but may include frequent and severe infections, splenomegaly, as well as other autoimmunity and inflammatory symptoms.
The approval was largely based on results of a 12-week randomized, placebo-controlled study in 31 patients with APDS (aged 12 years and older) who showed statistically significant improvement in the two primary endpoints of the trial – reduction in lymph node size and percentage of naïve B cells – when given leniolisib. The most common adverse reactions in the clinical trial were headache, sinusitis, and atopic dermatitis.
The approval also comes with a Priority Review Voucher to the developer of the drug, Pharming. Pursuant to the terms of Pharming’s 2019 exclusive license agreement with Novartis for the orphan drug leniolisib, Novartis has the right to purchase the voucher from Pharming for a small minority share of the value of the voucher.
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