Sanofi's oral BTK inhibitor rilzabrutinib received orphan drug designation in Japan for IgG4-related disease and warm autoimmune hemolytic anemia, while also securing U.S. FDA breakthrough therapy designation for the rare anemia indication.
Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to Sanofi's rilzabrutinib for IgG4-related disease, marking the third global orphan designation for this indication based on positive phase 2 study data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07549698 |
Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
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Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab |
RECRUITING | PHASE2 |
| NCT05925023 |
Sirolimus in the Treatment of Refractory/Relapsed wAIHA |
RECRUITING | NA |
| NCT05922839 |
Zanubrutinib in the Treatment of Relapsed/Refractory wAIHA |
RECRUITING | PHASE2 |
| NCT05786573 |
A Study of Obexelimab in Patients With Warm Autoimmune Hemolytic Anemia (SApHiAre) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05757570 |
An Open-label Study of Povetacicept in Participants With Autoimmune Cytopenias |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT05221619 |
Post-trial Access for Nipocalimab in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA) |
TEMPORARILY_NOT_AVAILABLE | |
| NCT04956276 |
Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia |
WITHDRAWN | PHASE2 |
| NCT04256148 |
ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia |
WITHDRAWN | PHASE2 |
| NCT04253236 |
To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA). |
TERMINATED | PHASE2 |
