Swedish Orphan Biovitrum (Sobi) and Apellis Pharmaceuticals have announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending the marketing authorisation of Aspaveli (pegcetacoplan) for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months.
The positive opinion from the CHMP is now referred to the European Commission for an approval decision.
“Today’s positive opinion by the CHMP is a significant milestone for people living with PNH across Europe,” says Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. “We hope to make a difference to the lives of people living with rare diseases, and if Aspaveli is approved by the European Commission, it will offer patients and treating physicians a new class of complement medicines for the treatment of PNH.”
Based on results from the PEGASUS study
The positive opinion is based on the results from the head-to-head phase 3 PEGASUS study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab.
Aspaveli is the European trade name for pegcetacoplan, which is known as Empaveli in the United States where it is approved for the treatment of adults with PNH.
Photo of Ravi Rao: Sobi