Pharming's Leniolisib Goes Under Priority FDA Review For Rare Immune System Disorder

The FDA has accepted Pharming Group NV's PHAR marketing application seeking approval for leniolisib for rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents.
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, aligned with a Priority Review classification.
Submitted by Pharming on July 29, 2022, the application was supported by positive data from a Phase 2/3 study of leniolisib, which met its co-primary endpoints of reduction in index lymph node size and correction of immunodeficiency in the target population.
Those results demonstrated the efficacy of leniolisib over placebo with a statistically significant reduction from the baseline size of participants' index lymphadenopathy lesions and normalization of their immune function.
Furthermore, the study's safety data showed that participants tolerated leniolisib well. Also submitted as part of the application were data from a long-term, open-label extension clinical trial, including 38 patients with APDS treated with leniolisib for 102 weeks.
Price Action: PHAR shares closed at $10.30 on Tuesday.