AstraZeneca's Oral PCSK9 Inhibitor AZD0780 Cleared for Phase III Trial by CDSCO Panel
New Delhi: The Subject Expert Committee (SEC) under the Central Drugs Standard Control Organisation (CDSCO) has granted approval to AstraZeneca Pharma India Limited to conduct a Phase III clinical trial for its investigational cardiovascular drug AZD0780.
The decision came during the 9th meeting of the SEC (Cardiovascular), held on 12th June 2025 at the CDSCO headquarters in New Delhi. During the meeting, the company presented the Phase III clinical study protocol (Protocol No. D7960C00015, Version 1.0 dated 11 April 2025) along with the Local CSP Addendum IND-1: Version 1.0 dated 14 April 2025.
AZD0780 is a novel oral PCSK9 inhibitor—a small molecule drug designed to reduce low-density lipoprotein cholesterol (LDL-C) levels. Unlike existing PCSK9 inhibitors such as alirocumab and evolocumab, which are injectable biologics (monoclonal antibodies), AZD0780 is administered orally, offering a potentially more convenient treatment option for patients with cardiovascular risk.
The drug works by binding to a specific pocket in the C-terminal domain of the PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) protein. This binding inhibits the interaction between PCSK9 and the LDL receptor (LDLR), thereby promoting the recycling of LDLRs to the cell surface. The result is enhanced clearance of LDL-C from the bloodstream, leading to lower cholesterol levels.
Following a detailed review, the expert panel found the trial design acceptable and recommended the grant of permission to proceed with the Phase III study as proposed by the company.
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