Pharming Group N.V. has announced the enrollment of the first patient in its Phase III clinical trial (NCT05438407) for leniolisib, aimed at treating children with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare genetic disorder. The trial will assess leniolisib's safety, efficacy, and tolerance in approximately 15 children aged 4 to 11 years across the U.S., Europe, and Japan. Primary efficacy endpoints include reducing lymph node size and increasing naïve B cell counts after 12 weeks. A subsequent trial for younger children (aged 1-6) is planned for Q3 2023. Leniolisib has shown promise in prior studies, and regulatory reviews are ongoing for broader approval.
Pharming Group N.V. (PHAR, PHGUF) announced that the European Medicines Agency (EMA) has validated its Marketing Authorisation Application (MAA) for leniolisib, an oral treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). The MAA, submitted in October 2022, will undergo an accelerated assessment, reducing the review period from 210 to 150 days. Positive Phase II/III study results showed leniolisib effectively reduced lymph node size and increased naïve B cell percentage in patients. Marketing authorisation is expected in H1 2023.