WAYRILZ (rilzabrutinib)
Wayrilz is a kinase inhibitor used for adults living with persistent or chronic immune thrombocytopenia (ITP). It helps patients who have already tried other therapies but did not see enough improvement in their condition. This medication provides an alternative option for managing long-term ITP by targeting the underlying immune processes that affect platelet levels.
How WAYRILZ Works
This drug works by targeting Bruton's tyrosine kinase (BTK) to modulate the immune system. It blocks B cell activation and stops the spleen and liver from destroying antibody-coated cells through the Fcγ receptor pathway. By decreasing autoantibody generation and signaling, it helps prevent the body from attacking its own platelets.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-08-29
- Patent Cliff
- 2041
- Revenue
- $7M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- TABLET
WAYRILZ Approval History
What WAYRILZ Treats
1 indicationsWAYRILZ is approved for 1 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Immune Thrombocytopenia
WAYRILZ Target & Pathway
ProTarget
A kinase essential for B-cell development and signaling. BTK inhibitors block B-cell receptor signaling, making them highly effective in B-cell malignancies like chronic lymphocytic leukemia and mantle cell lymphoma.
WAYRILZ Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in WAYRILZ's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications WAYRILZ treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to WAYRILZ
3 of 7FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
16 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06500702 RESULT | ACT18064 2024-511775-15, U1111-1301-2676 | Ph 2 | recruiting | A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease |
| NCT07190196 RILIEF | EFC17359 U1111-1316-0266, 2025-521398-15 | Ph 3 | recruiting | A 52-week Study of Rilzabrutinib Efficacy and Safety Compared to Placebo in Adults Diagnosed With IgG4-related Disease |
| NCT07086976 LUMINA 3 | EFC17360 U1111-1314-5112, 2024-517972-39 | Ph 3 | recruiting | A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia |
| NCT06975865 LIBRA | EFC17872 2024-518645-17, U1111-1311-1896 | Ph 3 | recruiting | The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease |
| NCT07007962 LUNA 4 | LPS18573 U1111-1320-4669 | Ph 3 | recruiting | Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment |
| NCT04562766 LUNA 3 results posted | EFC17093 PRN1008-018, 2023-509401-71 | Ph 3 | active not recruiting | Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) |
| NCT03395210 results posted | DFI17124 PRN1008-010, U1111-1260-4044 | Ph 2 | completed | A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP) |
| NCT07216079 | LTS19407 U1111-1319-5468 | Ph 3 | active not recruiting | Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan |
| NCT04520451 | ACT17125 PRN1008-017, U1111-1260-3972 | Ph 2 | completed | Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Participants |
| NCT05002777 LUMINA 2 | ACT17209 U1111-1262-2929, 2023-509441-13 | Ph 2 | active not recruiting | Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA) |
| NCT05107115 RILECSU | DRI17224 U1111-1263-4226, DRI17224 | Ph 2 | completed | Rilzabrutinib for the Treatment of Chronic Spontaneous Urticaria in Patients Who Remain Symptomatic Despite the Use of H1 Antihistamine |
| NCT05018806 | ACT17207 U1111-1261-7565, ACT17207 | Ph 2 | completed | Proof of Concept Study of Rilzabrutinib in Adult Patients With Moderate-to-severe Atopic Dermatitis |
| NCT06444204 | POP17091 U1111-1260-4098, PRN1008-020 | Ph 1 | completed | A Study to Evaluate the Effect of Mild and Moderate Hepatic Impairment on the Single-Dose Pharmacokinetics of Rilzabrutinib (PRN1008) |
| NCT06444191 | PKM17089 U1111-1260-4452, PRN1008-023 | Ph 1 | completed | Pharmacokinetics (PK) of Rilzabrutinib (PRN1008) in Healthy Japanese and Caucasian Subjects |
| NCT05104892 | ACT17208 U1111-1262-2956, 2021-002490-26 | Ph 2 | completed | Proof of Concept Study of Rilzabrutinib in Adult Participants With Moderate-to-severe Asthma |
| NCT03762265 results posted | EFC17092 PRN1008-012, 2018-002261-19 | Ph 3 | terminated | A Study of PRN1008 in Patients With Pemphigus |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
WAYRILZ FDA Label Details
Indications & Usage
FDA Label (PDF)WAYRILZ is indicated for the treatment of Immune Thrombocytopenia.
WAYRILZ Patents & Exclusivity
Patents (6 active)
Exclusivity
Pro Intelligence Preview
Deep insights for WAYRILZ
Revenue Insights
- • Q4-2025: $7M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2041
- • 7 active patents
Trial Analysis
- • 16 total trials
- • Stage: Expansion
Competitive Landscape
- • 7 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment