The PBS has listed iptacopan, an oral complement inhibitor, for adults with paroxysmal nocturnal haemoglobinuria (PNH), marking a major expansion of treatment options for the rare and life-threatening blood disorder.
The first-in-class factor B inhibitor was recommended for subsidy by the PBAC at its August 2024 meeting in the treatment of adults with PNH who have inadequate clinical response to C5 inhibitor treatment.
Effective 1 November 2025, iptacopan is now subsidised under the General Schedule as an immunosuppressant [link here], following earlier PBAC recommendations to accommodate switching between therapies including eculizumab, ravulizumab and pegcetacoplan.
The listing introduces four treatment phases – initial treatment, return from a PBS-subsidised complement 5 (C5) inhibitor, continuing therapy, and transitional “grandfather” arrangements for non-PBS patients – all subject to written authority application and specialist oversight.
Eligible patients must be aged 18 or over and have a PNH granulocyte clone size of at least 10% and either a haemoglobin level <105 g/L despite treatment with a C5 inhibitor for at least three months, or physician-confirmed intolerance requiring withdrawal. Treatment can only be prescribed by, or in consultation with, a haematologist, and must be the sole PBS-subsidised therapy for the condition.
The PBS cautions that iptacopan increases the risk of encapsulated bacterial infections, advising vaccination against meningococcal, pneumococcal and Haemophilus influenzae type B infection in accordance with the approved product information.
The authority application requires detailed monitoring data including haemoglobin, platelet and white-cell counts, reticulocytes, neutrophils, granulocyte clone size, and lactate dehydrogenase (LDH) ratios.
The move follows the June PBAC decision to recommend an expanded listing for pegcetacoplan (Empaveli), another targeted therapy for PNH. At the time, the committee noted that listing iptacopan would require flow-on changes to ensure switching options were available between all subsidised therapies, including eculizumab and ravulizumab.
“Cost-effectiveness would be acceptable if [pegcetacoplan] were cost-minimised against the long-acting C5 inhibitor, ravulizumab,” the PBAC stated in its March meeting outcome. It added that switching pathways would help manage intolerance or contraindications across the class.
Patients currently receiving iptacopan through non-PBS channels prior to 1 November are eligible to transition to subsidised therapy under grandfather arrangements. This provision is available once only, with ongoing access requiring enrolment under continuing treatment criteria. The grandfathering clause will expire 12 months from the date of listing.