Product Description
Leniolisib is an orally active, potent phosphatidylinositol 3-kinase inhibitor (PI3K) inhibitor, with selectivity for the PI3Kdelta isoform [2]. Novartis originally developed CDZ173 as a treatment for autoimmune diseases, but has rapidly repurposed it for its potential in patients with Activated PI3K Delta Syndrome (APDS, a.k.a. p110 delta activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency, or PASLI). APDS is a rare genetic immunodeficiency disease characterised by lymphoproliferation, recurrent infections from childhood, and an increased risk of EBV-associated lymphoma. APDS is known to be caused by autosomal dominant, gain-of-function mutations in the PIK3CD gene which encodes the PI3Kdelta protein [3,5]. (Sourced from: https://www.guidetopharmacology.org/GRAC/LigandDisplayForward?ligandId=9424)
Mechanisms of Action: PI3K Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Priority Review - *
Approval Status: Approved
Approved Countries: Czech | United States
Approved Indications: None
Known Adverse Events: None
Company: Pharming Technologies B.V.
Company Location:
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: France, Germany, Hungary, Japan, Portugal, Spain, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 15
Recent & Upcoming Milestones
- Leniolisib, a potential treatment for APDS in children, awaits FDA decision by January 31, 2026, following positive phase III results.
- Clinical Outcomes Reported - Pharming presented P3 Respiratory Tract Infections results on 2024-12-11 for Leniolisib
- PDUFA summary: Leniolisib's FDA goal date is March 29, 2023, for treating APDS, a rare immunodeficiency condition.
Highest Development Phases
Phase 3: Colitis|Lymphadenopathy|Other
Phase 2: Common Variable Immunodeficiency|Deficiency Diseases
Phase 1: Healthy Volunteers
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| LE 7201 | P2 |
Recruiting |
Deficiency Diseases |
2025-10-01 |
42% |
2024-11-13 |
Primary Completion Date|Primary Endpoints|Start Date|Study Completion Date|Treatments|Trial Status |
| LE 3301 | P3 |
Active, not recruiting |
Other |
2025-10-31 |
11% |
2025-10-29 |
Primary Endpoints |
| LE 4301 | P3 |
Recruiting |
Colitis |
2025-01-31 |
5% |
2024-02-09 |
Primary Endpoints |
| Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | P3 |
Completed |
Lymphadenopathy |
2025-01-30 |
35% |
2025-05-06 |
Primary Completion Date|Study Completion Date|Treatments|Trial Status |
| CVID | P2 |
Recruiting |
Common Variable Immunodeficiency |
2026-10-01 |
12% |
2025-03-27 |
Primary Endpoints|Treatments |
| jRCT2031230417 | P3 |
Recruiting |
Other |
2025-12-31 |
|||
| LE 3302 | P3 |
Active, not recruiting |
Other |
2025-12-23 |
2025-05-02 |
Treatments |
|
| LE 3301 | P3 |
Active, not recruiting |
Other |
2025-07-02 |
2025-05-02 |
Treatments |
|
| jRCT2061230036 | P3 |
Recruiting |
Unknown |
2025-04-30 |
|||
| LE 6101 | P1 |
Active, not recruiting |
Unknown |
2024-09-30 |
2025-05-02 |
Treatments |
|
| jRCT2031220625 | P3 |
Recruiting |
Other |
2024-10-31 |
|||
| LE 1102 | P1 |
Completed |
Healthy Volunteers |
2024-05-26 |
2025-05-02 |
Treatments |
|
| LE7X01 | P2 |
Recruiting |
Deficiency Diseases |
2028-09-18 |
12% |
2025-05-28 |
Primary Endpoints|Treatments |
| LE 8201 | P2 |
Not yet recruiting |
Common Variable Immunodeficiency |
2026-05-31 |
|||
| LE 3302 | P3 |
Active, not recruiting |
Other |
2026-07-09 |
11% |
2025-10-29 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |