The FDA has assigned a PDUFA goal date of March 29, 2023 for the NDA submission based on randomized-controlled and long-term extension data for leniolisib as a treatment for APDS, a rare primary immunodeficiency
LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ — Pharming Group NV (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM) (NASDAQ:PHAR) announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3 -kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023aligned with a Priority Review classification.
Submitted by Pharming on July 29, 2022the NDA was supported by positive data from a Phase II/III 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 (p=0.006) and normalization of their immune function, as evidenced by an increased proportion of naïve B cells from the baseline (p=0.002). Those findings indicate a reduction in disease markers associated with APDS, whose clinical hallmarks include significant lymphoproliferation and immune dysfunction, as well as increased risk of lymphoma. Furthermore, safety data from the study showed that leniolisib was well tolerated by participants. Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.
Anurag RelanMD, MPH, Chief Medical Officer of Pharming, commented:
“The FDA’s acceptance for priority review of Pharming’s New Drug Application for leniolisib is a milestone …