The expansion of the indication is based on data from APL2-308 which assessed Aspaveli over a period of 26 weeks compared to standard care measures
Sobi gets EC approval for Aspaveli to treat PNH. (Credit: Swedish Orphan Biovitrum AB)
Swedish Orphan Biovitrum (Sobi) said that the European Commission (EC) has approved an expanded indication of Aspaveli (pegcetacoplan) to treat naïve adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.
Aspaveli is a targeted C3 therapy designed to regulate the overactivation of the complement cascade, a part of the body’s immune system.
It serves as the European trade name for pegcetacoplan. The drug goes by the name Empaveli in the US, where it had gained approval for treating adults with PNH.
Apellis Pharmaceuticals handles its commercialisation in the US.
In Europe, the drug is approved for PNH adults who are anaemic after treatment with a C5 inhibitor for at least three months.
The expansion of the indication is based on data from APL2-308, an open-label, randomised, comparator-controlled study.
It recruited patients with PNH who had not undergone treatment with any complement inhibitor within three months before enrolment.
These individuals should also exhibit haemoglobin levels below the lower limit of normal, along with lactate dehydrogenase levels of ≥1.5 times the upper limit of normal.
The efficacy and safety of Aspaveli were assessed over 26 weeks compared to standard care measures.
Sobi chief medical officer and R&D and medical affairs head Lydia Abad-Franch said: “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.
“This important milestone reflects our dedication to improving treatment options for those affected by this rare and complex condition.”
The drug is also undergoing investigation for its potential efficacy in treating other rare diseases within the fields of haematology and nephrology.
The latest approval comes after Sobi received a positive Committee for Medicinal Products for Human Use (CHMP) opinion for efanesoctocog alfa in haemophilia A treatment.
CHMP recommended Efanesoctocog alfa’s approval for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A.