Sobi's Aspaveli approved for expanded use in Europe

The European Commission has granted Sobi approval for an extended indication of Aspaveli, a treatment for adults with paroxysmal nocturnal haemoglobinuria (PNH) who suffer from haemolytic anaemia. This chronic and potentially fatal blood disorder results from uncontrolled complement activation, which leads to the destruction of red blood cells responsible for oxygen transport.

In Europe, Aspaveli has been sanctioned for use in adult PNH patients who remain anaemic after undergoing treatment with a C5 inhibitor for a minimum of three months. Significantly, Aspaveli is now the first C3 inhibitor to receive approval for first-line PNH therapy in Europe. This approval has the potential to enhance patient outcomes by targeting haemoglobin levels alongside other clinical indicators.

The extended indication approval for Aspaveli is grounded in findings from the APL2-308 (PRINCE) study, which is an open-label, randomised, comparator-controlled clinical trial. The study included participants who had not been treated with any complement inhibitors for at least three months prior to enrolment. These patients displayed haemoglobin levels below the normal lower limit and lactate dehydrogenase levels at least 1.5 times above the normal upper limit.

Over a period of 26 weeks, the efficacy and safety of Aspaveli were evaluated in comparison to standard care treatments, which comprise interventions like blood transfusions and corticosteroids but exclude complement inhibitors.

Lydia Abad-Franch, Sobi's chief medical officer and head of research and development and medical affairs, noted: "Today’s approval underscores the robust clinical data supporting Aspaveli’s efficacy and safety profile, offering healthcare professionals and patients an expanded toolkit for effectively managing PNH. European patients will now be able to initiate treatment with Aspaveli at diagnosis or switch from their current C5 inhibitor treatment if they present indicators of haemolytic anaemia."

Aspaveli, known in the United States as Empaveli and commercialised by Apellis, is also approved there for the treatment of adults with PNH. Additionally, it is under investigation for its potential use in treating other rare diseases within the haematology and nephrology fields.

This expanded approval in Europe provides a new option for healthcare providers managing PNH, focusing on improving the quality of life and treatment outcomes for patients diagnosed with this debilitating condition.