Activated Pi3k-delta Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Novel Genetic Disorders of the Immune System
NIAID is conducting a clinical trial investigating novel genetic immune disorders, including PI3KCD, CTLA4, STAT3GOF, and MAGT1 deficiencies…
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CLINICAL_TRIALS trial_result · 2026-03-30Clinical Trial Update: Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI [Phase 2, Phase 3]
Terminated extension study (Phase 2/Phase 3) evaluating CDZ173 in Activated PI3K delta syndrome (APDS) and PASLI disease; results have been …
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CLINICAL_TRIALS research · 2026-03-23Clinical Trial Update: New Biomarker-based Strategy to Screen and Monitor for Activated Phosphoinositide 3-kinase δ Syndrome
A clinical trial is investigating a biomarker-based strategy to screen and monitor Activated PI3K delta syndrome, and the study is currently…
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CLINICAL_TRIALS research · 2025-10-28Clinical Trial Update: Pediatric Patients Aged 4 to 11 Years With APDS [Phase 3]
Phase 3 clinical trial evaluating a therapy for Activated PI3K-delta syndrome in pediatric patients aged 4 to 11 is currently active but not…
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CLINICAL_TRIALS research · 2025-10-28Clinical Trial Update: Pediatric Patients Aged 1 to 6 Years With APDS [Phase 3]
Phase 3 clinical trial evaluating a treatment for Activated PI3K-delta syndrome in pediatric patients aged 1 to 6 years is currently active …
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FDA approval · 2025-05-28FDA Approved: JOENJA — Supplemental Application (Pharming Healthcare Inc.)
FDA approved a supplemental application for Joenja (leniolisib) from Pharming Healthcare Inc., updating its regulatory approval status.
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CLINICAL_TRIALS trial_result · 2024-07-09Clinical Trial Update: Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome [Phase 1, Phase 2]
This document reports the completion of a Phase 1/2 trial evaluating rapamycin for Activated PI3K-delta syndrome (APDS), a primary immunodef…
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CLINICAL_TRIALS trial_result · 2024-02-08Clinical Trial Update: An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS [Phase 3]
Open-label Phase 3 trial evaluating safety and efficacy of leniolisib in Japanese patients with APDS (APDS gene mutation).
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FDA approval · 2023-03-24FDA Approved: JOENJA — Original Application (Pharming Healthcare Inc.)
FDA approved JOENJA (leniolisib) from Pharming Healthcare Inc. under NDA217759 for activated PI3K delta syndrome, establishing a first-in-cl…
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CLINICAL_TRIALS trial_result · 2021-06-18Clinical Trial Update: Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled Nemiralisib in Patients With APDS/PASLI [Phase 2]
Phase 2 safety, PK, and PD study of repeat doses of inhaled nemiralisib in patients with Activated PI3K-delta Syndrome has completed with re…