CDZ 173 given priority review by FDA for activated phosphoinositide 3-kinase delta syndrome
Pharming Group announced that the FDA has accepted for priority review its New Drug Application (NDA) for CDZ 173 (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kdelta) 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, 2023, aligned with a Priority Review classification.
Submitted by Pharming on July 29, 2022, the 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.
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