Drugs / Leniolisib
Leniolisib
also known as
CDZ173 · CDZ-173 FREE BASE · CDZ-173-AZ · CDZ-173-NX · Joenja · LENIOLISIB (CDZ 173) · Leniolisib monophosphate · LENIOLISIB PHOSPHATE
Small molecule targets PIK3CD via inhibition
Regulatory milestones approvals, filings & regulatory actions · 9 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed activated PI3K-delta syndrome 7 milestones | |||||
| Filed | US (FDA) | Joenja | activated PI3K-delta syndrome | 2026-09-25 | “FDA has accepted its supplemental New Drug Application (sNDA) seeking approval for lower doses of Joenja® (leniolisib)” pharming.com ↗ |
| Filed | US (FDA) | Joenja | activated PI3K-delta syndrome | 2026-06-04 | “FDA has accepted its resubmitted supplemental New Drug Application (sNDA) seeking approval for Joenja® (leniolisib)” pharming.com ↗ |
| Rejected (CRL) | US (FDA) | Joenja | activated PI3K-delta syndrome | 2026-01-30 | “FDA has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja® (leniolisib)” pharming.com ↗ |
| Filed | US (FDA) | Joenja | activated PI3K-delta syndrome | 2025-10-01 | “FDA has accepted its supplemental New Drug Application (sNDA) seeking approval for leniolisib” pharming.com ↗ |
| Filed | Japan (PMDA) | — | activated PI3K-delta syndrome | 2025-07-31 | “Submitted new drug application for leniolisib for the treatment of APDS in Japan” pharming.com ↗ |
| Filed | Other | — | activated PI3K-delta syndrome | 2023-08-03 | “efforts to make leniolisib available to APDS patients in key markets globally - CHMP opinion expected 4Q 2023, regulatory submissions filed in Canada, Australia, and Israel” pharming.com ↗ |
| Filed | US (FDA) | — | activated PI3K-delta syndrome | 2022-09-27 | “FDA has accepted for priority review its New Drug Application (NDA) for leniolisib” pharming.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Joenja | — | 2026-05-21 | europa.eu ↗ |
| Approved | US (FDA) | JOENJA | — | 2023-03-24 | fda.gov ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 24 trials · 1 missed | ||||||
| Phase 2 | NCT06990529 | Apr 2025 → Sep 2028 expected | activated PI3K-delta syndrome | Pharming Technologies B.V. | Recruiting | No outcome recorded |
| Phase 2 | NCT06897358 | Feb 2025 → Oct 2026 expected | common variable immunodeficiency | Pharming Technologies B.V. | Active not recruiting | No outcome recorded |
| Phase 2 | NCT06549114 | Oct 2024 → Nov 2026 expected | immunodeficiency disease | Pharming Technologies B.V. | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02775916 | Jun 2016 → May 2017 | Sjogren syndrome | Novartis Pharmaceuticals | Completed | Missed primary |
| Phase 2/32 trials · 1 met primary | ||||||
| Phase 2/3 | NCT02859727 | Sep 2016 → Jan 2025 | activated PI3K-delta syndrome | Pharming Technologies B.V. | Terminated | No outcome recorded |
| Phase 2/3 | NCT02435173 | Aug 2015 → Aug 2021 | activated PI3K-delta syndrome | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 33 trials | ||||||
| Phase 3 | NCT05693129 | Aug 2023 → Jul 2028 expected | activated PI3K-delta syndrome | Pharming Technologies B.V. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT06249997 | Aug 2023 → Jan 2025 | activated PI3K-delta syndrome | Pharming Technologies B.V. | Unknown | No outcome recorded |
| Phase 3 | NCT05438407 | Feb 2023 → Nov 2026 expected | activated PI3K-delta syndrome | Pharming Technologies B.V. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 21
| Date | Issuer | Release |
|---|---|---|
| 2026-09-11 | Pharming Technologies B.V. | Regulatory FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S. pharming.com ↗
Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib) |
| 2026-06-04 | Pharming Technologies B.V. | Regulatory Pharming announces U.S. FDA acceptance of sNDA resubmission for Joenja® (leniolisib) to treat children aged 4 to 11 years with APDS pharming.com ↗
We are pleased that the FDA has accepted our resubmission, bringing us one step closer to potentially making Joenja available to younger patients living with APDS. |
| 2026-05-22 | Pharming Technologies B.V. | Regulatory European Commission grants marketing authorization to Pharming’s Joenja® (leniolisib) – the first approved treatment for APDS in the European Union pharming.com ↗
Pharming Group (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) today announced that the European Commission (EC) has granted Marketing Authorization for Joenja® (leniolisib) |
| 2026-03-27 | Pharming Technologies B.V. | Regulatory Pharming Group receives positive CHMP opinion for Joenja® (leniolisib) for the treatment of APDS in adult and pediatric patients 12 years and older pharming.com ↗ |
| 2026-03-24 | Pharming Technologies B.V. | Regulatory Pharming Group announces approval of Joenja® (leniolisib) in Japan for the treatment of APDS in patients aged 4 years and older pharming.com ↗
Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS) in adult and pediatric patients aged 4 years and older. |
| 2026-02-01 | Pharming Technologies B.V. | Regulatory Pharming Group receives Complete Response Letter from U.S. FDA for sNDA for Joenja® (leniolisib) in children aged 4 to 11 years with APDS pharming.com ↗
Pharming Group (Euronext: PHARM; Nasdaq: PHAR) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. |
| 2025-10-01 | Pharming Technologies B.V. | Regulatory Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application for leniolisib in children with APDS aged 4 to 11 years pharming.com ↗
The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026. |
| 2025-04-23 | Pharming Technologies B.V. | Regulatory Pharming receives positive recommendation from NICE for Joenja®▼(leniolisib) as a treatment for APDS pharming.com ↗
Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the National Institute for Health and Care Excellence (NICE) has issued positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the National Health Service (NHS) in England and Wales |
| 2024-12-11 | Pharming Technologies B.V. | Results Pharming announces positive topline data in pediatric clinical trial of leniolisib pharming.com ↗
Data consistent with the improvements seen in the previously reported randomized controlled trial in adolescent and adult APDS patients |
| 2024-09-26 | Pharming Technologies B.V. | Regulatory Pharming announces marketing authorization in the U.K. for Joenja® (leniolisib) pharming.com ↗
Pharming Group N.V. announces that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. |
| 2024-05-30 | Pharming Technologies B.V. | Regulatory Pharming Group provides update on ongoing regulatory review of leniolisib for the treatment of APDS in the European Union pharming.com ↗
The CHMP requested that this work be completed pre-approval and has granted Pharming an extension to January 2026 to submit a response. |
| 2023-02-15 | Pharming Technologies B.V. | Regulatory Pharming provides update on EMA regulatory review of leniolisib for APDS in Europe pharming.com ↗
Pharming has received the list of questions from EMA which included a request to submit updated data from the ongoing long-term extension study collected after the interim analysis included in the original MAA. |
| 2022-12-06 | Pharming Technologies B.V. | Results Pharming announces publication of data from Phase 3 Study of leniolisib in patients with APDS in ASH’s Blood pharming.com ↗ |
| 2022-10-28 | Pharming Technologies B.V. | Regulatory Pharming Announces European Medicines Agency (EMA) Validates its Marketing Authorisation Application under Accelerated Assessment for leniolisib pharming.com ↗
EMA’s validation for review of our MAA under an accelerated assessment pathway highlights Pharming’s ongoing commitment to advance leniolisib as a targeted treatment for adults and adolescents 12 years of age or older with APDS. |
| 2022-10-10 | Pharming Technologies B.V. | Regulatory Pharming submits a Marketing Authorisation Application to the European Medicines Agency for Leniolisib pharming.com ↗
This MAA submission, under an accelerated regulatory pathway, is an important step towards approval of our second product in the EEA and highlights Pharming’s ongoing commitment to advancing leniolisib as a treatment for patients with APDS. |
| 2022-09-27 | Pharming Technologies B.V. | Regulatory Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib pharming.com ↗
Pharming Group N.V. announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. |
| 2022-08-01 | Pharming Technologies B.V. | Regulatory Pharming Group Receives Accelerated Assessment in Europe for leniolisib for the Treatment of Rare Immunodeficiency, APDS pharming.com ↗
The acceptance of an accelerated regulatory review for leniolisib underlines the high unmet need for patients with APDS, with the product potentially being the first approved treatment for this rare disease. |
| 2022-04-26 | Pharming Technologies B.V. | Regulatory Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA pharming.com ↗
The Company also announces that the MHRA has granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS. |
| 2022-03-31 | Pharming Technologies B.V. | Results Pharming Announces Positive Data from Phase II/III Leniolisib Trial Presented at Clinical Immunology Society 2022 Annual Meeting pharming.com ↗
It is great news that leniolisib has achieved such positive results in this Phase III study in APDS. |
| 2022-01-05 | Pharming Technologies B.V. | Regulatory Pharming receives positive EMA decision on paediatric investigation plan (PIP) for leniolisib in Europe pharming.com ↗
Pharming announces that a positive decision has been made by the European Medicines Agency (EMA) on the Paediatric Investigation Plan (PIP) for leniolisib, a phosphoinositide 3-kinase (PI3K) inhibitor, currently in development for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). |
| 2020-10-20 | Pharming Technologies B.V. | Regulatory Pharming receives Orphan Drug Designation from the European Commission for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) pharming.com ↗ |
All press releases naming this drug 39 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Leniolisib | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06549114 ↗ |
| Known as | CDZ-173 FREE BASE | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | CDZ-173-AZ | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | CDZ-173-NX | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | CDZ173 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02435173 ↗ |
| Known as | Joenja | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | LENIOLISIB (CDZ 173) | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | Leniolisib monophosphate | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Known as | LENIOLISIB PHOSPHATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL3643413 ↗ |
| Action | Inhibit | “an oral, small molecule inhibitor of PI3Kδ” PMID 36399712 ↗ Mar 2023 |
| Modality | Small molecule | “leniolisib, an oral, small molecule inhibitor of PI3Kδ” PMID 36399712 ↗ Mar 2023 |
| Route | Oral | “CDZ173 10 and 70 mg capsules for oral administration.” NCT02435173 ↗ |
| Target | PIK3CD | “a selective PI3K delta inhibitor” NCT02775916 ↗ |