Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib
Wednesday, September 28, 2022
Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for leniolisib, a selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, for priority review. Leniolisib is intended to treat primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents aged 12 and older in the US. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, granting it a Priority Review status.
The NDA, submitted by Pharming on July 29, 2022, was supported by positive data from a Phase II/III study of leniolisib. The study successfully achieved its co-primary endpoints, demonstrating a reduction in index lymph node size and correction of immunodeficiency in the target population. Leniolisib showed significant efficacy compared to the placebo, with a statistically significant reduction in the size of index lymphadenopathy lesions (p=0.006) and normalization of immune function, indicated by an increased proportion of naïve B cells from the baseline (p=0.002). These findings suggest a reduction in disease markers associated with APDS, which is characterized by lymphoproliferation, immune dysfunction, and an increased risk of lymphoma. The study also revealed that leniolisib was well tolerated by participants. Additionally, the NDA included data from a long-term, open-label extension clinical trial involving 38 APDS patients who received leniolisib treatment for a median duration of 102 weeks.
Dr. Anurag Relan, Chief Medical Officer of Pharming, expressed his perspective on the FDA's acceptance of leniolisib's NDA for priority review, highlighting its significance in addressing the unmet needs of patients with rare diseases. He emphasized that leniolisib has the potential to be a disease-modifying targeted treatment for APDS in adults and adolescents aged 12 and older in the US, who currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy. Pharming is committed to working closely with the FDA and regulatory authorities worldwide to make leniolisib accessible to immunologists, hematologists, and APDS patients.
