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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: 24 Aug 2024

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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

    • 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.
    • 03 Oct 2023 Interim results of an open-label extension study (between September 2016 and August 2021) assessing safety of Leniolisib for patients with Activated phosphoinositide 3-kinase delta syndrome, published in the Journal of Allergy and Clinical Immunology.

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