ITP refers to immune thrombocytopenia, a platelet disorder marked by low platelet counts and bleeding risk due to immune-mediated platelet destruction. It may be acute or chronic, with chronic disease more common in adults.
argenx reported $1.3B in Q1 2026 global product net sales (63% YoY growth). The FDA PDUFA target date for seronegative gMG is May 10, 2026. Positive ADAPT OCULUS results support an oMG label expansion. Pipeline milestones include myositis readout in Q3 2026 and MMN readout in Q4 2026.
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.
Novartis announced final Phase III ALIGN trial results showing Vanrafia (atrasentan) slowed kidney function decline in adults with IgA nephropathy, with a 2.39ml/min/1.73m² eGFR difference versus placebo at week 136.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07214025 |
Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children |
NOT_YET_RECRUITING | |
| NCT07194850 |
A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE2/PHASE3 |
| NCT07043946 |
A Phase 1b/2a Study of Budoprutug in Subjects With Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE1/PHASE2 |
| NCT05635266 |
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives |
RECRUITING | |
| NCT05446831 |
Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia |
UNKNOWN | PHASE2 |
| NCT05315778 |
Anti-BCMA CAR T-Cell Therapy for R/R ITP |
UNKNOWN | PHASE2 |
| NCT05281068 |
The Combination of Iguratimod and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia |
UNKNOWN | PHASE2 |
| NCT05118126 |
Gut Microbiota in ITP |
UNKNOWN | |
| NCT05116423 |
Machine-learning Based Prediction Model in Primary Immune Thrombocytopenia |
UNKNOWN | |
| NCT04943042 |
An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP |
COMPLETED |
