Also known as: Fabhalta
Iptacopan is an oral complement factor B inhibitor marketed as Fabhalta. It is used for paroxysmal nocturnal hemoglobinuria and primary IgA nephropathy, and later also approved in C3 glomerulopathy. The first FDA approval was in December 2023.
Novartis reported 2025 sales of $54.5 billion, up 8%, while earnings per share rose 15% to $8.98. Amarin reported fiscal 2025 revenue of $213.6 million, down 6.5%, and narrowed its net loss per share to $0.09 from $0.20.
Novartis commits $23 billion to U.S. manufacturing expansion with new facilities in North Carolina, California, and Florida, while advancing its immunoglobulin A nephropathy drug portfolio with positive Phase III results for Vanrafia.
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.
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 |
|---|---|---|---|
| NCT07347990 |
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy |
NOT_YET_RECRUITING | NA |
| NCT06994845 |
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN |
RECRUITING | PHASE3 |
| NCT06903234 |
Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry |
ACTIVE_NOT_RECRUITING | |
| NCT06847607 |
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA) |
NOT_YET_RECRUITING | NA |
| NCT06797518 |
Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN |
RECRUITING | PHASE2 |
| NCT06517758 |
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG. |
RECRUITING | PHASE3 |
| NCT06388941 |
Iptacopan in Patients With ANCA Associated Vasculitis |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05935215 |
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) |
RECRUITING | PHASE3 |
| NCT05755386 |
Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN |
RECRUITING | PHASE3 |
| NCT05630001 |
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. |
COMPLETED | PHASE3 |
