Paroxysmal nocturnal hemoglobinuria is a rare acquired clonal hematopoietic stem cell disorder caused by PIGA mutation leading to complement-mediated hemolysis, thrombosis risk, and marrow failure.
FDA granted traditional approval to Novartis' Fabhalta (iptacopan) for IgA nephropathy. Phase 3 APPLAUSE-IgAN data showed eGFR decline slowed by 48% versus placebo over two years.
China Medical System Holdings partnered with Insilico Medicine on AI-powered drug discovery in early February 2026 and received Chinese regulatory approval to begin human trials for complement factor B inhibitor CMS-D017.
Novartis announced final Phase III ALIGN results showing Vanrafia slowed kidney function decline in adults with IgA nephropathy, supporting planned submissions for traditional approval in 2026 following accelerated approvals in the U.S. and China in 2025.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07747090 |
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH. |
NOT_YET_RECRUITING | PHASE3 |
| NCT07657052 |
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria |
ENROLLING_BY_INVITATION | NA |
| NCT07470762 |
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria |
RECRUITING | PHASE1/PHASE2 |
| NCT07457151 |
Danicopan PMS in Korea |
RECRUITING | |
| NCT07416162 |
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy |
RECRUITING | |
| NCT07413679 |
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study |
ACTIVE_NOT_RECRUITING | |
| NCT07413250 |
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data |
ACTIVE_NOT_RECRUITING | |
| NCT07266155 |
Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
NOT_YET_RECRUITING | PHASE2 |
| NCT07177872 |
A Long-term Efficacy and Safety of NTQ5082 Capsules |
NOT_YET_RECRUITING | PHASE3 |
| NCT07177859 |
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients |
NOT_YET_RECRUITING | PHASE3 |
