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

 

• The FDA has accepted Pharming Group NV's 
  PHAR+0.97%+ Free Alerts
   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.

https://www.benzinga.com/general/biotech/22/09/29049343/pharmings-leniolisib-goes-under-priority-fda-review-for-rare-immune-system-disorder