CDSCO Panel Approves Novartis Healthcare’s Protocol Amendment to study Iptacopan
New Delhi: The Subject Expert Committee (SEC), functional under the Central Drug Standard Control Organization (CDSCO), has approved the protocol amendment proposal presented by Novartis Healthcare for Iptacopan (LNP023) study.
This came after the firm presented protocol amendment version 04 dated 30 Nov 2023, protocol no. CLNP023B12001B. This is an open-label, non-randomized extension study to evaluate the long-term efficacy, safety, and tolerability of iptacopan (LNP023) in C3 glomerulopathy or idiopathic immune-complex-membranoproliferative glomerulonephritis.
Glomerulopathy refers to any disease of the glomeruli, the tiny filters in the kidneys. Idiopathic immune-complex-membranoproliferative glomerulonephritis (IC-MPGN) is a specific type of glomerular disease where immune complexes and complement proteins are deposited in the kidney, potentially due to overactivation of the alternative complement pathway.
Iptacopan is a member of the class of indoles that is 1H-indole substituted by [(2S,4S)-2-(4-carboxyphenyl)-4-ethoxypiperidin-1-yl]methyl, methoxy, and methyl groups at positions 4, 5, and 6, respectively. It is a potent inhibitor of complement factor B with potential immunomodulatory activity. It has a role as a complement factor B inhibitor and an immunomodulator.
Iptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor B. Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase.
At the recent SEC meeting for Renal held on 18th March 2024, the expert panel reviewed the protocol amendment version 04 dated 30 Nov 2023 protocol no. CLNP023B12001B.
After detailed deliberation, the committee recommended approval of the protocol amendment as presented by the firm.