Basel, December 16, 2020 — Novartis today announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G).
The breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. The FDA granted BTD to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment8, as well as monotherapy in anti-C5 naïve PNH patients.
PNH is a rare and life threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function9,10, resulting in anemia, fatigue and other debilitating symptoms that can impact patients’ quality of life1–3. Despite current standard of care – anti-C5 therapy eculizumab or ravulizumab – a large proportion of PNH patients remain anemic and dependent on transfusions1,2,9,11,12.
FDA grants the rare pediatric designation for serious or life-threatening diseases primarily affecting individuals aged 18 years or younger and impacting fewer than 200,000 people. C3G, an ultra-rare and severe form of primary glomerulonephritis4,13, is characterized by complement dysregulation. It has a poor prognosis; about 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence post kidney transplant14. It has a worldwide annual incidence of 1-2 per million15.
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway6,7. It is currently in clinical development for PNH, as well as C3G and a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN).
Positive Phase II data from one study in PNH were presented at the European Society for Blood and Marrow Transplantation (EBMT) congress in August8, and Phase II interim analysis results in C3G were presented at the virtual 2020 Annual Meeting of the American Society of Nephrology (ASN) in October. Novartis is planning to initiate Phase III studies in several indications.
Iptacopan has the potential to become the first complement pathway inhibitor to slow disease progression in a number of complement-driven diseases. Based on disease prevalence and the positive interim data from Phase II studies, iptacopan has also received orphan drug designations from the FDA and EMA in C3G and PNH16, as well as EMA PRIME designation for C3G17, and EMA orphan drug designation in IgAN18.
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- Risitano AM and Rotoli B. Paroxysmal nocturnal hemoglobinuria: pathophysiology, natural history and treatment options in the era of biological agents. Biologics 2008;2(2):205–222.
- Hill A, et al. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica 2010;95(4):567–573.
- Schrezenmeier H, et al. Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry. Haematologica 2014;99(5):922–929.
- 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.
- 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.
- 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.
- Merle NS, et al. Complement system part II: role in immunity. Front Immunol 2015;6:257.
- 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 December 2020
- Hill A, et al. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers 2017;3:17028
- Risitano AM. Paroxysmal nocturnal hemoglobinuria and the complement system: recent insights and novel anticomplement strategies. Adv Exp Med Biol. 2013;735:155–72.
- Risitano AM. Anti-Complement Treatment in Paroxysmal Nocturnal Hemoglobinuria: Where we Stand and Where we are Going. Transl Med UniSa 2014;8:43–52.
- Debureaux P, et al. Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals. Blood 2019;134(Suppl 1):3517.
- Smith RJH, et al. C3 glomerulopathy – understanding a rare complement-driven renal disease. Nat Rev Nephrol. 2019;15(3):129–143.
- Nester CM and Smith RJ. Treatment options for C3 glomerulopathy. Curr Opin Nephrol Hypertens. 2013;22(2):231–237.
- Medjeral-Thomas NR, et al. C3 glomerulopathy: clinicopathologic features and predictors of outcome. Clin J Am Soc Nephrol. 2014;9(1):46–53.
- Novartis Data on File
- Novartis. Novartis received European Medicines Agency (EMA) PRIME designation for iptacopan (LNP) in C3 glomerulopathy (C3G). Available at: https://www.novartis.com/news/media-releases/novartis-received-european-medicines-agency-ema-prime-designation-iptacopan-lnp-c3-glomerulopathy-c3g. Accessed December 2020.
- Novartis. Novartis announces European Medicines Agency (EMA) has granted orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN). Available at: https://www.novartis.com/news/media-releases/novartis-announces-european-medicines-agency-ema-has-granted-orphan-drug-designation-iptacopan-lnp023-iga-nephropathy-igan. Accessed December 2020.
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