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 whom
etiologies of other types of TMA and non-aHUS kidney disease have been excluded.

- Currently on the recommended weight-based dosage regimen of anti-C5 antibody
treatment for at least 3 months prior to the screening visit.

- Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI)
for at least 3 months prior to entering the screening period as defined by:

1. Hematological normalization in platelet count ≥150 x 109/L and LDH below upper
limit of normal [ULN], and

2. Stable or improving kidney function as defined by ≤15% increase in serum
creatinine.

- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections
is required prior to the start of treatment with iptacopan.

- If not received previously or if a booster is required, vaccination against
Haemophilus influenzae infection, should be given, if available and according to
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 encapsulated
bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or
H. influenzae.

- Participants with sepsis or active systemic bacterial, viral (including COVID-19) or
fungal 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 solid
organ transplantation

- Female patients who are pregnant or breastfeeding, or intending to conceive during
the course of the study

- Any medical condition deemed likely to interfere with the patient's participation in
the study