An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.

Prior treatment

• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of study treatment and for 1 week after stopping study treatment. Effective contraception methods include: - Total abstinence (when this is in line with the preferred and usual lifestyle of the participant). Note that periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. - Bilateral oophorectomy with or without hysterectomy, total hysterectomy, or bilateral salpingectomy at least 6 weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment are they not considered to be of childbearing potential. - Bilateral tubal occlusion, bilateral tubal ligation (at least 6 weeks before taking study treatment).

Other illness

• 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 subject at increased risk or potentially confound study data.
• History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae.

Other

• Barrier methods of contraception: condom or occlusive cap (e.g., diaphragm or cervical/vault caps). For the UK: with spermicidal foam/gel/film/cream/vaginal suppository.
• Use of hormonal contraception methods: - Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation; oral, intravaginal or transdermal. - Progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable or implantable. - Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).

• Signed informed consent by parent(s)/legal guardian(s) for the patient must be obtained before enrolling in the roll-over study and receiving study medication. A consent or assent may also be required for some patients depending upon their age and local requirements.
• Male and female participant and has completed the treatment period in the Novartis sponsored pediatric study with iptacopan in any complement mediated disease (e.g., CLNP023I12201 or CLNP023G12301) on study drug.
• Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.
• Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e. any boosters required administered according to local regulations).
• Per investigator’s clinical judgement, the patient may benefit from continued treatment with iptacopan and has been clinically stable on iptacopan prior to enrollment in this extension study.