Novartis recently announced that the British Medicines and Healthcare Products Regulatory Agency (MHRA), the National Institute of Health and Clinical Optimization (NICE), and the Scottish Medical Federation (SMC) have awarded oral therapy iptacopan (LNP023) for the treatment of C3 glomeruli "Innovation Passport" qualification for disease (C3G)
.
C3G is an extremely rare and severe form of primary glomerulonephritis, characterized by a disorder of complement regulation
.
The annual incidence of the disease worldwide is 1-2 parts per million.
There are about 10,000 cases in the United States, about 10,500 cases in Europe, about 3,200 cases in Japan, and about 32,000 cases in China
.
C3G is usually diagnosed in adolescents and young adults.
The prognosis of the disease is very poor.
About 50% of patients develop end-stage renal disease (ESRD) within 10 years, and 50-70% of patients relapse after kidney transplantation
.
Iptacopan is a pioneering oral factor B inhibitor discovered by the Novartis Institute of Biomedical Research.
Factor B is a key serine protease in the alternative pathway of the complement system
.
Iptacopan has the potential to become the first targeted therapy to delay the progress of C3G dialysis
.
The Innovation Passport is a pass to the Innovation Permit and Access (ILAP)
.
ILAP is a new way to accelerate the approval of drugs launched after Brexit.
It was launched in January 2021.
It aims to accelerate the approval and marketing of innovative drugs and promote patients' access to drugs
.
ILAP provides a single integrated platform for collaboration between MHRA, medical partners including NICE and SMC, and medical developers
.
Drug developers under this channel will receive more feedback from MHRA and stakeholders (including NICE)
.
A single innovation passport can cover multiple future indications for the same active substance
.
Therefore, drugs can also involve multiple diseases in the subsequent steps of ILAP
.
iptacopan is a first-in-class, oral, potent, selective, small molecule, and reversible factor B inhibitor.
Factor B is a key serine protease in the alternative pathway of the complement system
.
Currently, iptacopan is being developed to treat many complement-driven renal diseases (CDRD) with significant unmet medical needs, including C3G, IgA nephropathy, atypical hemolytic uremic syndrome (aHUS), membranous nephropathy (MN) ), and the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH)
.
The published Phase 2 data showed that: (1) treatment of IgAN with iptacopan reduced proteinuria and stabilized renal function; (2) treatment of C3G reduced the rate of decline of estimated glomerular filtration rate (eGFR) and stabilized renal function; (3) Treatment of PNH significantly reduces intravascular and extravascular hemolysis, enabling most patients to achieve rapid and long-lasting improvement without blood transfusion
.
Although Novartis has 35 years of history in the treatment of kidney transplantation, iptacopan is the first treatment for CDRD in the kidney disease treatment pipeline
.
Novartis’s goal is to change the treatment approach by targeting one of the key drivers of these rare and progressive diseases, which has the potential to extend the non-dialysis life of CDRD patients
.
Note: The original text has been deleted
Original source: Novartis Pharmaceuticals UK awarded an Innovation Passport for investigational oral therapy iptacopan (LNP023)
