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An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor Blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 (Leniolisib) in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/ p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

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Trial Profile

An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor Blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 (Leniolisib) in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/ p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

Status: Completed
Phase of Trial: Phase II/III

Latest Information Update: 30 Apr 2025

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At a glance

  • Drugs Leniolisib (Primary)
  • Indications Common variable immunodeficiency; Immunodeficiency disorders; Lymphadenopathy
  • Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Registrational; Therapeutic Use
  • Sponsors Novartis; Novartis Pharmaceuticals

Most Recent Events

  • 23 Apr 2025 According to Pharming Group NV media release, Based on data from this trial,the National Institute for Health and Care Excellence has issued positive final guidance recommending Joenja for reimbursement and use within the National Health Service in England and Wales for the treatment of activated phosphoinositide 3 kinase delta syndrome in adult and pediatric patients 12 years of age and older
  • 01 Aug 2024 According to Pharming Group NV media release, On March 12, 2024, Pharming submitted an MAA for leniolisib with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), through the International Recognition Procedure (IRP) on the basis of the U.S. FDA approval. The MAA for leniolisib was validated on April 17, 2024. Pharming received the MHRA Day 70 Request for Further Information on July 3, 2024. There were no major objections. Upon Pharming's satisfactory response to MHRA requests.
  • 30 May 2024 According to Pharming Group NV media release, the CHMP review is ongoing and following the May 27-30 CHMP meeting, Pharming received an updated List of Outstanding Issues from the CHMP. The LoOI affirmed the positive clinical benefit and safety of leniolisib, in agreement with the assessment by the Ad Hoc Expert Group, and included one remaining CMC. The CMC request relates to the definition of regulatory starting materials used in the manufacturing of leniolisib.

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