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Home Novartis Granted UK Innovation Passport for Iptacopan, a First-in-Class Oral Factor B Inhibitor Targeting the Root Cause of Complement-Mediated Diseases

Novartis Granted UK Innovation Passport for Iptacopan, a First-in-Class Oral Factor B Inhibitor Targeting the Root Cause of Complement-Mediated Diseases

Oct 31, 2021 22:10 CST Updated 22:10
Novartis

Drug Development and Manufacturing

Medicines and Healthcare products Regulatory Agency

The MHRA is an executive agency of the UK Department of Health and Social Care, responsible for ensuring that medicines and medical devices work effectively and are safe and reliable. Established in 2003 through the merger of the Medicines Control Agency and the Medical Devices Agency, the organization employs more than 1,300 staff members.

NICE

NICE is a non-departmental public body of the UK Department of Health, primarily responsible for: National Health Service, clinical practice of health technologies, guidelines for health promotion and disease prevention, and social care services. It serves the UK NHS.

Scottish Medicines Consortium

The Scottish Medicines Consortium (SMC) provides advice to NHS Scotland on the patient value of newly licensed medicines. The SMC is part of Healthcare Improvement Scotland. We review new medicines that have been licensed by the Medicines and Healthcare products Regulatory Agency (MHRA, the UK licensing authority). We also review new formulations and new uses of established medicines. Before a medicine can be routinely prescribed in Scotland, it must be accepted for use by the SMC. Our advice helps health services plan for the rapid and consistent introduction of beneficial treatments across NHS Scotland.

Kidney (Image source: parashospitals.com)

October 31, 2021 /BioonBIOON/ --Novartis(Novartis) recently announced that the UK medicines and`` `Dietary Supplements` ``The Medicines and Healthcare products Regulatory Agency (MHRA), the National Institute for Health and Care Excellence (NICE), and the Scottish Medicines Consortium (SMC) have granted"Innovation Passport" designation for oral iptacopan (LNP023) for the treatment of C3 glomerulopathy (C3G)。

C3G is an extremely rare and severe primary glomerulonephritis characterized by complement dysregulation. The disease has a global annual incidence of 1 to 2 per million, with approximately 10,000 cases in the United States, 10,500 in Europe, 3,200 in Japan, and 32,000 in China. C3G typically occurs in adolescents and young adults and is usuallyDiagnosisCome out,This disease carries a very poor prognosis; approximately 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience recurrence following kidney transplantation.

Iptacopan isNovartisA first-in-class oral factor B inhibitor discovered by a biomedical research institute; 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 progression to dialysis in C3G.

The Innovation Passport serves as the gateway to the Innovative Licensing and Access Pathway (ILAP).。ILAP is a new regulatory pathway for accelerated drug approval introduced following Brexit, launched in January 2021, and designed to expedite the regulatory approval and market availability of innovative medicines while improving patient access to treatments.. ILAP provides a single integrated platform for collaboration among the MHRA, healthcare partners (including NICE and SMC), and pharmaceutical developers. Under this pathway, medicine developers will receive more feedback from the MHRA and stakeholders (including NICE). A Single Innovation Passport can cover multiple future indications for the same active substance. Consequently, the medicine may also address multiple diseases in subsequent stages of the ILAP.

Chemical Structure of Iptacopan (Image source: medchemexpress.com)

Iptacopan is a first-in-class, oral, potent, selective, small-molecule, reversible factor B inhibitor, where factor B is a key serine protease in the alternative pathway of the complement system.

Currently, iptacopan is being developed for the treatment of many complement-driven renal diseases (CDRD) with significant unmet medical needs., including C3G, IgA nephropathy, atypical hemolyticUremiaatypical hemolytic uremic syndrome (aHUS), membranous nephropathy (MN), and the rare hematologic disorder paroxysmal nocturnal hemoglobinuria (PNH).

Published Phase 2 data show:（1）Iptacopan treatment for IgAN reduced proteinuria and stabilized renal function; (2) treatment for C3G slowed the rate of decline in estimated glomerular filtration rate (eGFR) and stabilized renal function; (3) treatment for PNH significantly reduced intravascular and extravascular hemolysis, enabling rapid and sustained transfusion-free improvement in the majority of patients.

Although Novartis has a 35-year history in kidney transplantation, iptacopan is the first therapy in its nephrology pipeline to address CDRD.NovartisThe objective is to transform the treatment paradigm by targeting one of the key drivers of these rare and progressive diseases, which has the potential to extend dialysis-free survival for patients with CDRD.

NovartisChinmay Bhatt, Managing Director for the UK, Ireland and the Nordics at Novartis, said: “Ensuring our medicines reach the patients who need them most is a key priority for us. We are proud to have developed a medicine that has been granted an Innovative Passport under the new Innovative Licensing and Access Pathway. We are delighted to engage with stakeholders across the UK healthcare system through this new route to explore opportunities to bring this innovative medicine to patients as soon as possible.” (Bioon.com)