Pegcetacoplan Clinical Trial (Phase 3) Results Published
A recent article in the prestigious journal ′”New England Journal of Medicine” published the findings of the phase 3 study of a C3 inhibitor (pegcetacoplan or APL2), intended to be used to treat PNH. Eculizumab and ravulizumab medications are C5 inhibitors. C3 and C5 are proteins which are part of the complement system (complement system: the congenital immune system of the body) and the drugs interrupt the complement cascade which attacks the PNH affected blood cells. This study compared the C3 inhibitor Pegcetacoplan (or APL2) to eculizumab and found that Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients by providing broad hemolysis control, including control of intravascular and extravascular hemolysis. C5 inhibitors such as Eculizumab and ravulizumab don’t control extravascular hemolysis. Marketing applications (which licence drugs to be used) for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted this application Priority Review designation and set a target action date of May 14, 2021. An opinion from the EMA Committee for Medicinal Products for Human Use (CHMP) is expected in 2021. Pending licensing, the NICE process to appraise Pegcetacoplan for payment (for use in adults with PNH whose anaemia is not controlled after treatment with a C5 complement inhibitor) has begun and PNH Support has been invited to make submissions by 24 May 2021. If you have been involved in an APL2 (Pegcetacoplan) trial, please get in touch on contact@pnhuk.org. Unfortunately the full publication is behind a paywall but we are trying to obtain a plain language summary. https://www.nejm.org/doi/full/10.1056/NEJMoa2029073…