Sobi’s C3 therapy, Aspaveli (pegcetacoplan), has been approved by the European Commission (EC) as a first-line treatment for paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.
PNH is a rare and severe blood disorder in which uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells. The condition is characterised by persistently low haemoglobin and can result in frequent transfusions and symptoms such as severe fatigue caused by anaemia.
Despite improvements with C5 inhibitor treatment, cross-sectional surveys conducted in the US and EU show that up to 86% of PNH patients treated with C5 inhibitors remain anaemic.
Aspaveli, which is designed to regulate excessive activation of the complement cascade, is already approved in Europe to treat adults with PNH who are anaemic following treatment with a C5 inhibitor for at least three months.
The EC’s latest authorisation is supported by data from the APL2-308 study, which enrolled PNH patients 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 of at least 1.5 times the upper limit of normal.
Lydia Abad-Franch, head of research and development and medical affairs, and chief medical officer at Sobi, said the approval “underscores the robust clinical data supporting Aspaveli’s efficacy and safety profile” and offers 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,” Abad-Franch added.
Pegcetacoplan is approved to treat PNH as Aspaveli or Empaveli in the EU, US and other countries globally, and the therapy is also being investigated in other rare diseases across haematology and nephrology.
Sobi has exclusive commercialisation rights for systemic pegcetacoplan outside the US, where Apellis Pharmaceuticals has exclusive rights. Apellis also holds global commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
The approval comes just days after Sobi and Sanofi’s efanesoctocog alfa was recommended for approval by the European Medicines Agency’s human medicines committee as a once-weekly treatment for the blood clotting disorder haemophilia A.
