European Commission approves an indication extension for Aspaveli (pegcetacoplan) for treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia

Aspaveli is already approved in Europe for the treatment of adults with PNH who are anaemic after treatment with a C5 inhibitor for at least three months.

Aspaveli is now the first C3 inhibitor approved for first-line treatment of PNH in Europe, offering effective outcomes by improving haemoglobin and other clinical markers due to its unique mode of action.

PNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells. Characterised by persistently low haemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anaemia. Despite improvements with C5 inhibitor treatment, up to 86 per cent of people with PNH treated with C5 inhibitors remain anaemic, according to cross- surveys conducted in US and EU.

The indication extension is based on data from APL2-308 (PRINCE, NCT04085601), an open-label, randomised, comparator-controlled study that enrolled patients with PNH who had not been treated with any complement inhibitor within three months prior to enrolment and with haemoglobin levels less than the lower limit of normal and lactate dehydrogenase levels greater than 1.5 times the upper limit of normal. Efficacy and safety of Aspaveli was evaluated over a duration of 26 weeks compared to standard of care (e.g., transfusions, corticosteroids, supplements such as iron, folate, and vitamin B12; excluding complement inhibitors).

Aspaveli is the European trade name for pegcetacoplan, which is known as Empaveli in the United States where it is also approved for the treatment of adults with PNH and commercialised by Apellis.