Adults in Scotland with rare blood disorder to get access to new oral monotherapy on NHS
January 13, 2025Novartis said in a press release Monday that the Scottish Medicines Consortium (SMC) has published advice recommending Fabhalta (iptacopan) for use on the NHS in Scotland as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.
Iptacopan can only be used under the advice of the National PNH Service, according to the announcement.
PNH is a rare blood disease where red or white blood cells are vulnerable to being attacked by a particular part of the body’s immune system called “the complement system”5-8.The process by which red blood cells are destroyed is called haemolysis and is responsible for many PNH symptoms, including fatigue.8,9 Up to 80% of patients with PNH experience fatigue caused by anaemia, having a direct impact on quality of life and it means they can also require blood transfusions.
Lesley Loeliger, Founder and Chair of PNH Scotland said: “The announcement by the SMC that iptacopan is to be made available in Scotland is good news for patients and families living with PNH. The option of an oral monotherapy for patients with PNH and haemolytic anaemia is exciting. We would like to thank the SMC for allowing the patient voice to be heard during the evaluation of medicines for rare diseases.”
Dr Morag Griffin, Consultant Haematologist at Leeds Teaching Hospitals NHS Trust said: “We at the National PNH service are pleased with the approval of iptacopan as the first oral monotherapy for people living with PNH in Scotland. Trial data has shown iptacopan can be effective in improving haemoglobin levels and controlling the destruction of red blood cells that causes many of the symptoms of PNH. I look forward to working with the team at University Hospital Monklands to get this treatment to eligible patients”
The decision was based on the APPLY-PNH and APPOINT-PNH Phase III clinical trials. The APPLY-PNH study compared iptacopan with C5 inhibitor therapies in adult patients with PNH presenting with persistent anaemia despite a stable regimen of C5 inhibitor therapies. The APPOINT-PNH study was a single arm study of iptacopan in adult PNH patients who are naïve to C5 inhibitor therapies, Novartis said.
Johan Kahlström, Country President Novartis UK and Ireland, said: “We were pleased to secure access to the first oral monotherapy for PNH patients in England and Wales in September, and it’s great that iptacopan is now an option for patients in Scotland too. At Novartis, we are continuing to demonstrate how committed we are to delivering potentially practice changing treatments in rare diseases.”