NICE publish final guidance recommending Aspaveli in adults with ultra-rare blood disorder

The drug is additionally undergoing assessment with the Medicines and Healthcare products Regulatory Agency for a marketing authorisation in the UK.

Sobi UK announced that the National Institute for Care Excellence (NICE) has issued the final appraisal decision recommending the use of Aspaveli (pegcetacoplan) in adults with paroxysmal nocturnal haemoglobinuria (PNH) in England and Wales. PNH is a chronic, progressive, debilitating and life-threatening blood disorder.

The guidance published, following a fast track appraisal by NICE, recommends Aspaveli as an option for treating adults with PNH who are anaemic after treatment with a C5 inhibitor for at least three months. The guidance follows results from the head-to-head phase 3 PEGASUS study, which evaluated the efficacy and safety of pegcetacoplan compared to eculizumab.

“Pegcetacoplan is a valuable step forwards in treatment for patients with PNH. Despite recent treatment advances, some patients with PNH who are being treated with the current standard of care experience low haemoglobin levels and extravascular haemolysis, causing anaemia and fatigue. This adversely affects their quality of life and can lead to blood transfusion requirement,” said Dr Morag Griffin, consultant haematologist at Leeds Teaching Hospitals NHS Trust.

PNH affects approximately 900 people in England. It is characterised by hemolysis (destruction of red blood cells) that is mediated by an uncontrolled activation of the complement system, part of the body’s immune system. PNH can result in frequent blood transfusions and debilitating symptoms including severe fatigue, as in 96% of cases and anaemia in 88% of cases. If left untreated, it has a 10-year mortality rate of 29% with 40-67% of deaths caused by thrombosis.

“Until now, real world evidence studies indicate that many patients receiving treatment continue to experience debilitating symptoms, such as anaemia-related fatigue, which significantly impact their quality of life, and result in them having to adjust to a ‘new normal’,” shared Michael Oliver, general manager at Sobi UK & Rol. “This recommendation paves the way for an alternative treatment option which can help tackle this unmet need.”