?The European Medicines Agency has granted iptacopan a priority medicines (PRIME) designation in C3 glomerulopathy (C3G).
?PRIME is granted for medicines that may offer major therapeutic advance or benefit patients without treatment options.
?C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need.1–3
?Iptacopan (LNP023) is a potential first-in-class, oral, potent and selective factor B inhibitor of the complement system’s alternative pathway, targeting the underlying cause of C3G. 4–6

Basel, October 9, 2020 — Novartis today announced that the European Medicines Agency (EMA) has granted PRIME designation for iptacopan (LNP023) in C3 glomerulopathy (C3G). PRIME is a program launched by the EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary program is based on enhanced interaction and early dialogue with developers of promising medicines, to optimize development plans and speed up evaluation so these medicines can potentially reach patients earlier.

C3G is an ultra-rare and severe form of primary glomerulonephritis, characterized by complement dysregulation.1,7 It has a worldwide annual incidence of 1–2 per million8 and an approximate prevalence of 10,000 in the US, ~10,500 in Europe, 3,200 in Japan and 32,000 in China9.

C3G is commonly diagnosed in adolescents and young adults. The disease has a poor prognosis; about 50% of patient progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence post kidney transplant.2

Results from a Phase II interim analysis for iptacopan in C3G will be presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting from October 22-25, 2020.

About iptacopan
Iptacopan is a first-in-class oral, small-molecule, reversible inhibitor of factor B, a key serine protease of the alternative pathway of the complement cascade.4,5

In addition to C3G, iptacopan is in parallel development for a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy (IgAN), atypical hemolytic uremic syndrome and membranous nephropathy.

Novartis is also investigating iptacopan in paroxysmal nocturnal hemoglobinuria (PNH). Following positive Phase II data presented at the European Society for Blood and Marrow Transplantation (EBMT) congress in August10, a randomized, active-comparator controlled open-label Phase III trial to evaluate the efficacy and safety of iptacopan in PNH patients with residual anemia despite treatment with anti-C5 antibody therapy is planned to start in Dec 2020.11

Iptacopan has the potential to become the first alternative pathway inhibitor to slow disease progression in a number of complement driven diseases. Based on the preliminary data from the ph2 trial, Iptacopan has received orphan designations by FDA and EMA in C3G.

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References

1. Martín B and Smith R. C3 Glomerulopathy. [Last Update: April 5, 2018]. Available at: https://www.ncbi.nlm.nih.gov/books/NBK1425?. Accessed September 2020.
2. Nester CM and Smith RJ. Treatment options for C3 glomerulopathy. Curr Opin Nephrol Hypertens. 2013;22(2):231–237.
3. Goodship TH, et al. Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) Controversies Conference. Kidney Int. 2017;91(3):539–551.
4. Merle NS, et al. Complement System Part II: Role in Immunity. Front Immunol. 2015;6:257.
5. Schubart A, et al. Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci U S A. 2019;116(16):7926–7931.
6. Harris, C. L. Expanding horizons in complement drug discovery: challenges and emerging strategies. Semin Immunopathol. 2018;40(1):125–140.
7. Smith RJH, et al. C3 glomerulopathy - understanding a rare complement-driven renal disease. Nat Rev Nephrol. 2019;15(3):129–143.
8. Medjeral-Thomas NR, et al. C3 glomerulopathy: clinicopathologic features and predictors of outcome. Clin J Am Soc Nephrol. 2014;9(1):46–53.
9. DRG Epidemiology Report, August 2019.
10. Novartis. Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH). Available at: https://www.novartis.com/news/media-releases/novartis-announces-positive-results-from-phase-ii-study-lnp023-patients-paroxysmal-nocturnal-hemoglobinuria-pnh. Accessed September 2020.
11. Novartis Data on File

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