Leniolisib granted priority review for activated phosphoinositide 3-kinase delta syndrome

The FDA granted priority review to a new drug application for leniolisib to treat activated phosphoinositide 3-kinase delta syndrome in adults and adolescents aged 12 years and older, according to a Pharming Group press release.

Approximately one to two people per million are affected by activated phosphoinositide 3-kinase delta syndrome (APDS), which is caused by variants in two genes that regulate the maturation of white blood cells.

Leniolisib, an oral, selective phosphoinositide 3-kinase delta inhibitor, inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which Pharming Group called an important cellular messenger that activates AKT and regulates multiple cell functions.

The new drug application was supported by a phase 2/3 study, during which patients on leniolisib experienced a statistically significant reduction in their index lymphadenopathy lesions from baseline (P = .006) compared with patients on placebo. Also, patients on leniolisib vs. placebo saw normalized immune function based on their increased proportion of naïve B cells from baseline (P = .002). The drug was well tolerated.

These findings indicate that this targeted treatment for APDS reduces disease markers associated with the disease, according to the press release.

“The FDA’s acceptance for priority review of Pharming’s new drug application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases,” Anurag Relan, MD, MPH, chief medical officer of Pharming Group, said in the release.

The FDA assigned the new drug application for leniolisib a prescription drug user fee act target date of March 29, 2023.