Novartis Gets CDSCO Panel Nod to study Iptacopan

Iptacopan is a factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria. 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.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that presents clinically with a variety of symptoms, the most prevalent of which are hemolytic anemia, hemoglobinuria, and somatic symptoms including fatigue and shortness of breath.

Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, the generation of downstream effectors, and the amplification of the terminal pathway.

In paroxysmal nocturnal hemoglobinuria, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization. Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated EVH and terminal complement-mediated IVH.

At the recent SEC meeting for Cardiovascular and Renal held on 6th and 7th December 2023, the expert panel reviewed the drug major Novartis's proposal to conduct the trial for the drug Iptacopan.

After detailed deliberation, the committee recommended for grant of permission to conduct the trial as presented by the firm with the condition that diagnosis of Membranoproliferative glomerulonephritis (MPGN) should be done from a single nephropathologist by doing light microscopy, IF, and electron microscopy.

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