Study design for FABHALTA^® (iptacopan)

IN ADULTS WITH C3G, TO REDUCE PROTEINURIA¹

• Study design¹: A randomized, double-blind, placebo-controlled study (up to Month 6)

• Study population¹: 74 adults with biopsy-confirmed native kidney C3G who had a UPCR ≥1 g/g and eGFR ≥30 mL/min/1.73 m².  Safety and effectiveness of FABHALTA in patients with recurrent C3G following kidney transplant have not been established

• Background therapy¹: Patients were required to be on a maximally tolerated RASi and could be on a corticosteroid and/or MMF/MPS at baseline. All background therapies (eg, RASi, MMF/MPS, corticosteroids) were required to be at stable doses for 90 days prior to randomization and throughout the study. Randomization was stratified according to whether patients were receiving concomitant immunosuppressive therapy

^*Defined as a ≥50% reduction in 24-hour UPCR compared to baseline and stable or improved eGFR compared to baseline (≤15% reduction in eGFR).¹

KEY INCLUSION CRITERIA^1,2

• UPCR ≥1 g/g

• eGFR or measured GFR ≥30 ml/min/1.73 m²

• Serum C3 <77 mg/dL

• Patients aged ≥18 to ≤60 years with biopsy-confirmed native kidney C3G in the 12 months prior to enrollment

• On a stable, maximally tolerated RASi. All background therapies (eg, RASi, corticosteroids, and MMF/MPS) were required to be at stable doses for 90 days

• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae. Recommendation to be vaccinated against Haemophilus influenzae type b at least 2 weeks prior to first dose

KEY EXCLUSION CRITERIA²

• Solid-organ or cell transplant, including kidney transplant

• Rapidly progressive crescentic glomerulonephritis

• Renal biopsy showing interstitial fibrosis/tubular atrophy of more than 50%

• Postinfectious glomerulonephritis

• Monoclonal gammopathy

• Active systemic bacterial, viral, or fungal infection or the presence of fever

• History of recurrent invasive infections caused by encapsulated organisms

• Use of complement inhibitors within 6 months prior to screening

• Use of immunosuppresants (except MMF), cyclophosphamide, or systemic corticosteroids (prednisolone >7.5 mg/day or equivalent) within 90 days of randomization

Click on image to enlarge.