0%). 2%). 9%). The most commonly reported serious adverse reaction was pneumococcal infection (1%). Tabulated list of adverse reactions Table 1 shows the adverse reactions observed in the clinical studies with iptacopan in patients with PNH and C3G.
Adverse reactions are listed by MedDRA system organ class (SOC) and frequency, using the following convention: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1 000 to <1/100), rare (≥1/10 000 to <1/1 000) or very rare (<1/10 000).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness. 7 Table 1 Adverse reactions System Organ Class Adverse reaction Frequency category PNH C3G Infections and infestations Upper respiratory tract infection1 Very common Very common Urinary tract infection2 Common Bronchitis3 Common Pneumococcal infection4 Common Pneumonia bacterial Uncommon Blood and lymphatic system disorders Platelet count decreased Common Nervous system disorders Headache5 Very common Dizziness Common Gastrointestinal disorders Diarrhoea Very common Abdominal pain6 Common Nausea Common Skin and subcutaneous tissue disorders Urticaria Uncommon Musculoskeletal and connective tissue disorders Arthralgia Common 1 Upper respiratory tract infection includes preferred terms influenza, nasopharyngitis, pharyngitis, rhinitis, sinusitis, upper respiratory tract infection, and viral upper respiratory tract infection.
2 Urinary tract infection includes preferred terms urinary tract infection and cystitis escherichia. 3 Bronchitis includes preferred terms bronchitis, bronchitis haemophilus and bronchitis bacterial. 4 Pneumococcal infection includes preferred terms pneumonia pneumococcal and pneumococcal sepsis.
5 Headache includes preferred terms headache and head discomfort. 6 Abdominal pain includes preferred terms abdominal pain, abdominal pain upper, abdominal tenderness and abdominal discomfort. 6%) patients with PNH reported serious bacterial pneumonia while receiving treatment with iptacopan; the patient had been vaccinated against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type B and recovered following treatment with antibiotics while continuing treatment with iptacopan.
In C3G completed clinical studies, 1 patient with C3G reported serious pneumococcal infection with pneumonia and sepsis while receiving treatment with iptacopan; the patient had been vaccinated against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type B and recovered following treatment with antibiotics.
Iptacopan treatment was interrupted and restarted after recovery. Platelet count decreased in patients with PNH Decrease in platelet count events was reported in 12/164 (7%) patients with PNH. Of these, 5 patients had events of mild severity, 5 had moderate events and 2 had severe events.
Patients with severe events had concurrent anti-platelet antibodies or idiopathic bone marrow aplasia with pre-existing thrombocytopenia. The events started within the first 2 months of iptacopan treatment in 7/12 patients, and after a longer exposure (111 to 951 days) in 5/12 patients.
At the cut-off date, 7 (58%) patients had recovered or events were resolving and iptacopan treatment was continued throughout in all patients. 7 mmol/l were seen at month 6 for total cholesterol and LDL-cholesterol. The mean values remained within the normal ranges.
7 mmHg at month 6). The mean DBP did not exceed 80 mmHg. 1). In patients treated with iptacopan 200 mg twice a day in the C3G clinical study, no clinically relevant differences were observed in total cholesterol, LDL-cholesterol or blood pressure compared to placebo.
Heart rate decrease in patients with PNH In patients treated with iptacopan 200 mg twice a day in PNH clinical studies, a mean decrease in heart rate of approximately 5 bpm was seen at month 6 (mean of 68 bpm). Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important.
It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.