New medicine for rare diseases! C3 inhibitor pegcetacoplan received FDA priority review: Phase 3 efficacy beats heavyweight C5 inhibitor Soliris

November 18, 2020 // -- Apellis Pharma is a leader in the development of C3 targeted therapies and is committed to pioneering and best-in-class therapies through a ground-breaking 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 the U.S. Food and Drug Administration (FDA) has accepted and granted priority review to a new drug application (NDA) for Pgcetacoplan (APL-2), a drug used to treat cystic sleep hemoglobinuria (PNH), which has been shown in clinical trials to raise PNH standards and hopefully redefine PNH treatment.
fda has designated a target date for the Prescription Drug User Charge Act (PDUFA) as May 14, 2021.
fda said it did not plan to convene an advisory committee meeting to discuss the NDA.
previously, the FDA had granted pegcetacoplan fast-track eligibility (FTD) for PNH treatment.
currently under review by the European Medicines Agency (EMA) for marketing authorization (MAA) for the treatment of PNH.
"For more than a decade, the only option for treating PNH has been C5 inhibitors, but many patients still experience persistent hypohemoglobin, which often leads to weak fatigue and frequent blood transfusions," said Dr. Federico Grossi, chief medical officer of apellis.
FDA's granting of NDA priority review brings us one step closer to providing pegcetacoplan to patients in need, a targeted C3 therapy that has the potential to redefine PNH treatment.
data from the NDA confirm the broad potential of targeting C3, and we are continuing to advance several registered studies of serious diseases with little or no treatment options.
"Pegcetacoplan's NDA and MAA are based on the results of the head-to-head Phase 3 PEGASUS Study (NCT03500549) (see: PEGASUS Phase 3 Top-Line 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 is as secure as Soliris.
(click on the image: see the graph) it's worth noting 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 United States, 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 C5 proteins 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 rare and super rare diseases.
Soliris was first approved for 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 spinal cord lineal 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 aHUS for children and adult patients.
Ultomiris is the first and only long-acting C5 supplement inhibitor given every 8 weeks, and in a Phase III clinical study treating PNH, Ultomiris infusions every 2 months (8 weeks) and Soliris infusions every 2 weeks achieved non-poor effectiveness at all 11 endpoints.
() Original source: Apellis Announces FDA Acceptance and Priority Review of of the New Drug application for Pegcetacoplan for the Treatment of PNH