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Paroxysmal nocturnal haemoglobinuria (PNH) Clinical criteria: Treatment Phase: Initial treatment (new patient) Patient must not have received prior treatment with this drug for this condition, AND Patient must have PNH granulocyte clone size equal to or greater than 10% within the last 3 months, AND Patient must have experienced an inadequate response/intolerance to either: (i) Complement 5 (C5) inhibitor, (ii) pegcetacoplan, AND Patient must have received treatment with at least one complement inhibitor for at least 3 months before initiating treatment with this drug unless intolerance of severity necessitating permanent treatment withdrawal had occurred, AND The treatment must be the sole PBS-subsidised therapy for this condition. Treatment criteria: Must be treated by a haematologist; OR Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details. Population criteria: Patient must be at least 18 years of age. The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include: (1) details of the proposed prescription(s); and (2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice). At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided: (i) Haemoglobin (g/L) (ii) Platelets (x109/L) (iii) White Cell Count (x109/L) (iv) Reticulocytes (x109/L) (v) Neutrophils (x109/L) (vi) Granulocyte clone size (%) (vii) Lactate Dehydrogenase (LDH) (viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory (ix) the LDH:ULN ratio (in figures, rounded to one decimal place)
“Iptacopan is a proximal complement inhibitor that targets Factor B (FB) to selectively inhibit the alternative pathway while leaving the direct signalling from the lectin and classical pathways intact.”
“The half-life (t1/2) of iptacopan at steady state is approximately 25 hours after administration of FABHALTA 200 mg twice daily.”
Working under the parallel aged-care framework? Aged-care equivalent →
Curated subset. The full adverse-effect list is in the TGA Product Information; click any citation above to open it.
“Metabolism of iptacopan includes N-dealkylation, O-deethylation, oxidation, and dehydrogenation, mostly driven by CYP2C8 (98%) with a small contribution from CYP2D6 (2%).”