Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Inclusion Criteria:

• Male and female participants age ≥ 12 and ≤ 60 years at screening.

• Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollmentin adults and within 3 years in adolescents.

• Prior to randomization, all participants must have been on a maximally recommendedor tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) orangiotensin receptor blocker (ARB) for at least 90 days. The doses of otherantiproteinuric medications including mycophenolic acid, corticosteroids andmineralocorticoid receptor antagonists should be stable for at least 90 days priorto randomization.

• Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratorynormal range) at Screening.

• UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.

• Estimated GFR (using the CKD-EPI formula for ages ≥ 18 years and modified Schwartzformula for ages 12 to 17 years) or measured GFR ≥ 30 ml/min/1.73m2 at screening andDay -15.

• Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniaeprior to the start of study treatment.

• If not previously vaccinated or if a booster is required, vaccination againstHaemophilus influenzae infections should be given, if available and according tolocal regulations, at least 2 weeks prior to the first study treatmentadministration. If study treatment has to start earlier than 2 weeks postvaccination, prophylactic antibiotic treatment should be initiated.

Exclusion Criteria:

• Participants who have received any cell or organ transplantation, including a kidneytransplantation.

• Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in theeGFR within 3 months with renal biopsy findings of glomerular crescent formationseen in at least 50% of glomeruli.

• Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%

• Monoclonal gammopathy of undetermined significance (MGUS) confirmed by themeasurement of serum free light chains or other investigation as per local standardof care.

• Participants with an active systemic bacterial, viral or fungal infection within 14days prior to study treatment administration

• The presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatmentadministration.

• A history of recurrent invasive infections caused by encapsulated organisms, e.g.,N. meningitidis and S. pneumoniae.

• The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3inhibitors, anti C5 antibodies, C5a receptor antagonists) within 6 months prior tothe Screening visit.

• The use of immunosuppressants (except mycophenolic acids), cyclophosphamide orsystemic corticosteroids at a dose >7.5 mg/day (or equivalent for a similarmedication) within 90 days of study drug administration.

• Acute post-infectious glomerulonephritis at screening based upon the opinion of theinvestigator.