Five intra-articular gene therapy platforms for knee osteoarthritis are in human trials in the US, while India prepares regulatory guidelines for mRNA, gene and cell therapies to accelerate innovation.
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 |
|---|---|---|---|
| NCT07569081 |
A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome |
NOT_YET_RECRUITING | PHASE2/PHASE3 |
| NCT07542314 |
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting |
NOT_YET_RECRUITING | PHASE4 |
| NCT07518277 |
A Multicenter, Prospective, Randomized Controlled Study Comparing Glucocorticoid Combined With Sirolimus With Monotherapy of Glucocorticoid in the Treatment of Newly Diagnosed Mild Autoimmune Hemolytic Anemia |
NOT_YET_RECRUITING | NA |
| NCT07340463 |
The Efficacy and Safety of Biologics (Belimumab/ Telitacicept) Induction Therapy in Proliferative Lupus Nephritis Patients for 6 Months Compared With Mycophenolate Mofetil Treatment |
RECRUITING | PHASE2 |
| NCT07152366 |
Efficacy and Safety of Sequential Hormone Therapy and Tetuzumab Therapy in Patients With Moderate to Severe TAO in the Active Stage After Glucocorticoid Treatment. |
RECRUITING | PHASE4 |
| NCT07152340 |
The Safety and Efficacy of Sequential Hormone Therapy and IBI311 Therapy in Patients With Active Moderate to Severe TAO in the Initial Treatment. |
RECRUITING | PHASE4 |
| NCT06957002 |
Bosentan in the Treatment of Giant Cell Arteritis |
NOT_YET_RECRUITING | PHASE2 |
| NCT06904040 |
A Single-center Study of CM313 in Patients With Pemphigus |
NOT_YET_RECRUITING | NA |
| NCT06868290 |
Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA |
RECRUITING | PHASE2 |
| NCT06673134 |
Prospective Cohort Establishment and Clinical Observation of Children With Crohn's Disease |
ACTIVE_NOT_RECRUITING |
