Target C3 nephropathy (C3G) the underlying cause! Novart's powerful selective factor B inhibitor iptacopan is eligible for EU priority drugs!

Oct 11, 2020 // -- Novartis recently announced that the European Medicines Agency (EMA) has granted iptacopan (LNP023) priority drug eligibility (PRIME) for the treatment of C3 nephropathy (C3G).
PRIME is a rapid approval program launched by EMA in March 2016, similar to the FDA's Breakthrough Drug Qualification (BTD) program, designed to accelerate the review process of key drugs in the field of pharmaceutical shortages and benefit patients as early as possible.
's finalists, PRIME's experimental drugs, will receive strong support from the EMA in clinical trials and drug development to accelerate the development and approval of truly innovative drugs to meet the medical needs of promising new drugs.
a PRIME-qualified drug, there must be preliminary clinical evidence and non-clinical evidence that the drug substantially improves the condition compared to existing therapeutic drugs.
iptacopan is a pioneering (first-in-class), oral, powerful, selective, small molecule, reversible B-factor inhibitor, B-factor is the key to the replacement pathway of the complement system serine protease.
iptacopan can target the underlying cause of C3 nephropathy (C3G).
C3G is an extremely rare and serious primary ngnephritis characterized by a rehydration disorder.
the annual incidence of the disease worldwide is 1-2 per million, about 10,000 in the United States, about 10,500 in Europe, about 3,200 in Japan and about 32,000 in China.
C3G is usually diagnosed in adolescents and young adults, and the prognosis is poor, with about 50% of patients developing end-stage kidney disease (ESRD) within 10 years and 50-70% relapsing after kidney transplantation.
will present the results of the interim analysis of the phase II study of iptacopan treatment for C3G at the 2020 annual meeting of the American Society of Nephrology (ASN) from October 22 to 25, 2020.
addition to C3G, iptacopan is also being developed in parallel to treat a number of other kidney diseases that are suffering from adjunct systems and have significantly unsolvent needs, including IgA nephropathy (IgAN), atypical hemolytic uremia syndrome, and membrane nephropathy.
addition, Novaral is also studying the efficacy of iptacopan in treating hairy sleep-related hemoglobinuria (PNH).
Based on Phase II positive data presented at the European Society for Blood and Bone Marrow Transplant (EBMT) conference on August 10, Novarma plans to begin a randomized, positive drug-controlled, open-label Phase III trial in December 2020 to assess the efficacy and safety of iptacopan in patients with PNH (despite receiving anti-C5 antibody therapy).
in many supplement-driven diseases, iptacopan has the potential to be the first alternative pathway inhibitor to slow disease progression.
based on preliminary data from Phase 2 trials, iptacopan has been granted ODD status by the FDA and the European Union's EMA for the treatment of C3G orphan drugs.
() Origin: Novartis received European Medicines Agency (EMA) PRIME Designation for Iptacopan (LNP) in C3 glomerulopathy (C3G)