Novartis’ First-in-Class Factor B Inhibitor Iptacopan Granted EMA PRIME Designation for Targeting Root Cause of C3 Glomerulopathy

October 11, 2020 News /BioValleyBIOON/ --Novartis(Novartis) recently announced that the European Medicines Agency (EMA) has granted iptacopan (LNP023) Priority Medicines (PRIME) status for the treatment of C3 glomerulopathy (C3G).

PRIME is a fast-track approval program launched by the EMA in March 2016, similar to that of the United StatesFDAsimilar to the Breakthrough Therapy Designation (BTD) program, aims to accelerate the review process for key medicines in areas of unmet medical need, thereby enabling patients to benefit as early as possible. Investigational drugs selected for the PRIME scheme will beClinical Trialsand has received strong support from the EMA in drug development to accelerate the development and approval of truly innovative medicines, thereby addressing the medical need for promising new drugs. To qualify for PRIME designation, a medicine must have preliminary clinical and non-clinical evidence demonstrating that it can provide a substantial improvement over existing treatments.

Iptacopan is a first-in-class, oral, potent, selective, small-molecule, reversible Factor B inhibitor. Factor B is a key serine protease in the alternative pathway of the complement system. Iptacopan targets the root cause of C3 glomerulopathy (C3G).

C3 glomerulopathy (C3G) is an extremely rare and severe form of primary glomerulonephritis characterized by dysregulation of the complement system. The annual incidence worldwide is 1–2 cases per million population, with approximately 10,000 cases in the United States, 10,500 in Europe, 3,200 in Japan, and 32,000 in China.C3G is usually found in adolescents and young adults.DiagnosisThe prognosis of this disease is poor, with approximately 50% of patients progressing to end-stage renal disease (ESRD) within 10 years, and 50-70% experiencing recurrence after kidney transplantation.。

NovartisAmerican Society of Nephrology (ASN) 2020 Annual Meeting, to be held from October 22 to 25, 2020MeetingInterim Analysis Results of the Phase II Study of Iptacopan in the Treatment of C3G

Kidneys (Image source: parashospitals.com)

In addition to C3G, iptacopan is also being developed in parallel for the treatment of other kidney diseases involving the complement system with significant unmet needs, including IgA nephropathy (IgAN) and atypical hemolyticUremiasyndrome, membranous nephropathy. Furthermore,NovartisAlso investigating the efficacy of iptacopan in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Based on the positive Phase II data presented at the European Society for Blood and Marrow Transplantation (EBMT) Congress on August 10,NovartisA randomized, active-controlled, open-label Phase III trial was planned to commence in December 2020 to evaluate the efficacy and safety of iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) despite treatment with anti-C5 antibodies.

In many complement-driven diseases, iptacopan has the potential to become the first alternative pathway inhibitor that slows disease progression. Based on preliminary data from Phase 2 trials, iptacopan has been approved by the U.S.FDAand the orphan drug designation (ODD) granted by the EU EMA for the treatment of C3G. (Bioon.com)

Original Source: Novartis received European Medicines Agency (EMA) PRIME designation for iptacopan (LNP) in C3 glomerulopathy (C3G)