Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Inclusion Criteria:

• Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whometiologies of other types of TMA and non-aHUS kidney disease have been excluded.

•. Currently on the recommended (as per label) dosage regimen of anti-C5 antibodytreatment, for at least 3 months prior to entering the screening period.

• In the opinion of the investigator the participant has responded to anti-C5antibodytreatment prior to screening and has clinical evidence of response (inabsence of PE/PI) during the Screening period.

Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:

1. Hematological normalization in platelet count ≥150 x 10^9/L and LDH below upperlimit of normal [ULN], and

2. Stable kidney function as defined by serum creatinine values within ±15% during theScreening period

• Vaccination against Neisseria meningitidis and Streptococcus pneumoniaeinfections is required prior to the start of treatment with iptacopan.

• If not received previously or if a booster is required, vaccination againstHaemophilus influenzae infection, should be given, if available and accordingto local regulations.

Exclusion Criteria:

• History of aHUS disease relapse while on anti-C5 antibody treatment.

• eGFR < 30 ml/min/1.73m^2

• Active infection or history of recurrent invasive infections caused by encapsulatedbacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) orH. influenzae.

• Participants with sepsis or active systemic bacterial, viral (including COVID-19) orfungal infection within 14 days prior to study treatment administration.

• Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT),heart, lung, small bowel, pancreas, liver transplantation or any other cell or solidorgan transplantation

• Female patients who are pregnant or breastfeeding, or intending to conceive duringthe course of the study

• Any medical condition deemed likely to interfere with the patient's participation inthe study