New medicine for rare diseases! C3 inhibitor pegcetacoplan U.S. and European applications for listing: head-to-head phase 3 efficacy beats heavyweight C5 inhibitor Soliris.

16, 2020 // -- Apellis Pharmaceuticals is committed to developing pioneering and best-in-class therapies through a groundbreaking targeted C3 approach to treat a wide range of diseases driven by uncontrolled or overactivated complement cascading reactions, including hematology, ophthalmology and nephrology.
recently, the company announced that it had submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval for pegcetacoplan (APL-2) for the treatment of cystic sleep haemoglobinuria (PNH).
FDA and EMA will make a decision on the acceptance of the NDA and MAA in the fourth quarter of 2020.
in clinical trials, pegcetacoplan has demonstrated its potential to raise PNH care standards.
submitted by the NDA and MAA is based on the results of the head-to-head Phase 3 PEGASUS Study (NCT03500549) (see: PEGASUS Phase 3 Top-Line Results Conference Call Presentation).
The study reached its main endpoint, demonstrating that pegcetacoplan was superior to the PNH standard care drug Soliris (eculizumab): hemoglobin levels improved statistically at 16 weeks, the normalization rate of key markers of hemolysis was higher, and the FACIT-fatigue score was clinically significant.
in this study, pegcetacoplan was as secure as Soliris.
noted that pegcetacoplan was the first research therapy to show superior hemoglobin levels over Soliris, with up to 85 percent of patients treated with pegcetacoplan not receiving blood transfusions.
of PNH patients currently treated with Soliris suffer from persistent anemia.
PEGASUS study showed that pegcetacoplan has the potential to become the new standard of care for PNH patients.
pegcetacoplan is a research, targeted C3 inhibitor designed to regulate overactivation of supplements, which is the cause of many serious diseases.
pegcetacoplan is a synthetic cyclopeptide that binds to a polyethyl glycol polymer and specifically binds to C3 and C3b.
, pegcetacoplan is being developed to treat a variety of diseases, including PNH, map-like atrophy (GA) and C3 renal pelagic disease.
, the FDA has granted fast-track eligibility for pegcetacoplan treatments for PNH and GA.
Isoliris is a drug sold by Alexion, a pioneering supplement inhibitor that works by inhibiting the C5 protein in the end part of the complement cascading reaction terminal.
cascading response is part of the immune system, and its uncontrolled activation played an important role in a variety of serious and super-rare diseases.
Soliris was first approved for the market in 2007 and has previously been approved for a variety of ultra-rare diseases: PNH, atypical hemolytic uremia syndrome (aHUS), anti-AchR antibody-positive systemic severe muscle weakness (gMG), anti-water channel protein-4 (AQP4) antibody-positive visual neurospinal spectrum disorder (NMOSD).
is one of the world's best-selling orphanages, with sales of $3.563 billion in 2018.
, Alexion is also developing an upgraded version of Soliris' Ultomiris, which was approved by the FDA in December 2018.
October 2019, Ultomiris was approved by the FDA for a new adaptation: the treatment of children and adults with aHUS.
Ultomiris was the first and only long-acting C5 supplement inhibitor given every 8 weeks, and in a Phase III clinical study treating PNH, Ultomiris was non-effective in terms of infusions every 2 months (8 weeks) and Soliris every 2 weeks at all 11 endpoints.
() Original origin: Apellis Announces Submission of Pegcetacoplan Marketing Applications to FDA and EMA for Patients with PNH.