Novartis' iptacopan shows promising action in rare renal disease

• Novartis (NYSE:NVS) reports positive Phase II interim analysis results for iptacopan (LNP023), an investigational oral treatment for C3 glomerulopathy (C3G), a rare renal disease, affecting young patients with a poor prognosis.
• Data from the study showed that after 12 weeks, iptacopan significantly reduced proteinuria by 49% compared to baseline values in 12 patients (P=0.0005).
• Iptacopan strongly inhibited alternative complement pathway activity and improved plasma C3 levels.
• In addition, iptacopan stabilized renal function as assessed by eGFR (estimated glomerular filtration rate) at week 12, and this effect was maintained in the seven patients treated into the long-term extension study.
• Iptacopan also had a favorable safety and tolerability profile, with no deaths and no serious adverse events leading to treatment discontinuation.
• The EMA has granted PRIME designation for iptacopan in C3G.
• The data was presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting.
• Additionally, on October 23, the EMA granted an Orphan Drug designation for iptacopan (LNP023) in IgA nephropathy, following a recommendation from the Committee for Orphan Medicinal Products. The status provides a 10-year period of market exclusivity for the indication in Europe, if approved.
• On October 21, Novartis secured Orphan Drug designation for branaplam (LMI070) in Huntington’s disease (HD) in the U.S. Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.
• https://seekingalpha.com/news/3625582-novartis-iptacopan-shows-promising-action-in-rare-renal-disease