- 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.
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