ЄС схвалив Аспавелі: лікування пароксизмальної нічної гемоглобінурії (ПНГ)

Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB (Sobi) recently announced that the European Commission (EC) has approved Aspaveli (pegcetacoplan) for the treatment of paroxysmal sleep caused by anemia after receiving C5 inhibitors for at least 3 months. Adult patients with sexual hemoglobinuria (PNH). In May 2021, the drug was the first to be approved in the United States under the trade name Empaveli. It can be used for adult PNH patients who have not previously been treated, and it can also be used for PNH adult patients who have switched treatments from C5 inhibitors (Soliris and Ultomiris). The approval of pegcetacoplan for marketing is expected to improve the standard of PNH care and redefine the treatment of PNH.

It is worth mentioning that pegcetacoplan is the first and only C3 targeted therapy to obtain regulatory approval. In the past decade or so, the only option for the treatment of PNH was C5 inhibitors, but many patients still experience persistent hypohemoglobinopathy, often leading to weak fatigue and frequent blood transfusions. In clinical trials, pegcetacoplan can provide extensive control of PNH, improving the lives of PNH patients by increasing hemoglobin levels and reducing the need for blood transfusions.

pegcetacoplan has obtained regulatory approvals in the United States and the European Union, based on the results of a head-to-head phase 3 PEGASUS study (NCT03500549) (see: PEGASUS Phase 3 Top-Line Results Conference Call Presentation). The study reached the primary endpoint, demonstrating that pegcetacoplan is better than Soliris, the PNH standard of care drug: it is better than Soliris in the change of hemoglobin level from the baseline level at the 16th week, and the adjusted hemoglobin level increased by 3.84g/dL on average (p<0.0001)>

In addition, pegcetacoplan achieved non-inferiority in avoiding blood transfusion endpoints compared to Soliris. 85% of patients in the pegcetacoplan treatment group had no blood transfusion within 16 weeks, compared with 15% in the Soliris treatment group. At the same time, compared with patients in the Soliris treatment group, patients in the pegcetacoplan treatment group have a higher normalization rate of key hemolysis markers, and the FACIT-fatigue score has a clinically significant improvement. In this study, pegcetacoplan is as safe as Soliris.

pegcetacoplan is the first therapy to show superior hemoglobin levels than Soliris, and up to 85% of patients treated with pegcetacoplan do not have a blood transfusion. Most of the PNH patients currently receiving Soliris treatment suffer from persistent anemia. The results of the PEGASUS study show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.

Пегцетакоплан – це цільовий інгібітор С3, призначений для регулювання надмірної активації комплементу, що є причиною виникнення та розвитку багатьох серйозних захворювань. Pegcetacoplan – це синтетичний циклічний пептид, який зв’язується з полімером поліетиленгліколю і специфічно зв’язується з C3 і C3b. Зараз пегцетакоплан розробляється для лікування різноманітних захворювань, включаючи ПНГ, географічну атрофію (ГА) та гломерулопатію С3. У Сполучених Штатах FDA надав pegcetacoplan швидку кваліфікацію для лікування ПНГ та ГА.

Currently, there are no drugs to treat GA. Apellis plans to submit a new drug application for intravitreal injection of pegcetacoplan to treat age-related macular degeneration (AMD) secondary GA in 2022.