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
Single group
Iptcaopan 200 mg
DrugOpen label, participant specific kits, hard gelatin capsules to be taken twice a day
Number of participants with adverse events and serious adverse events
Number of participants with adverse events and serious adverse events, including abnormal safety laboratory parameters, vital signs and ECGs
Number of participants with absence of TMA manifestation without the use of anti-C5 antibody
Thrombotic microangiopathy (TMA) manifestation is defined by the coexistence of at least two of the following three criteria at the same visit attributable to aHUS: * thrombocytopenia (platelet count decrease of ≥ 25% compared to baseline and \< lower limit of normal (LLN)), * microangiopathic hemolytic anemia (hemoglobin ≤ LLN for age and gender and lactate dehydrogenase (LDH) ≥ 1.5 x upper limit of normal (ULN), * worsening kidney function (serum creatinine increase of ≥ 25% compared to baseline levels)
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
Complete thrombotic microangiopathy (TMA) Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 109/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline or ≥ 25% serum creatinine reduction compared to serum creatinine values prior to initiation of anti-C5 antibody therapy)
Estimated glomerular filtration rate (eGFR)
Estimated glomerular filtration rate (eGFR) based on eGFR categories will be collected. Serum creatinine as measured in mg/dL as part of the clinical chemistry panel through the central laboratory will be used to calculate the eGFR applying the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
Chronic kidney disease (CKD) stage
Chronic kidney disease (CKD) stage (1-5) based on eGFR categories will be provided: * Stage 1 (G1): Kidney damage with normal kidney function * Stage 2 (G2): Mild loss of kidney function * Stage 3 (G3): 3a: Mild to moderate loss of kidney function; 3b: Moderate to severe loss of kidney function * Stage 4 (G4): Severe loss of kidney function * Stage 5 End stage renal disease (kidney failure): Kidney failure and need for transplant or dialysis
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