This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
Signed informed consent must be obtained prior to participation in the open label extension study
Willing and able to comply with the study Schedule of Activities
Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301 , CLNP023F12302), are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement
Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines)
Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk
Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes