A Complete Response Letter (CRL) has been issued by the Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for inclisiran, an investigational treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy.
In the letter, the FDA stated that the application could not be approved in its current form due to “unresolved facility inspection-related conditions” and that “satisfactory resolution of the unresolved facility inspection-related conditions is required before the Novartis NDA may be approved.”
Inclisiran is a chemically synthesized small interfering RNA that targets proprotein convertase subtilisin-kexin type 9 (PCSK9) messenger RNA, thereby preventing the production of the target protein in the liver, increasing hepatic uptake of LDL-C and clearing it from the bloodstream. The subcutaneous injectable is being investigated for its ability to lower LDL-C through twice-a-year dosing. Findings from the ORION clinical development program have shown clinically meaningful and sustained reductions in LDL-C with twice-yearly dosing following an initial dosing regimen on day 1 and month 3.
Commenting on the CRL, John Tsai, Head Global Drug Development and Chief Medical Officer, Novartis, said: “Novartis is confident in the quality of the regulatory submission for inclisiran, which includes a robust body of evidence related to efficacy and safety. We look forward to meeting with the FDA and our third-party manufacturing partner to discuss the feedback received and next steps.”
For more information visit novartis.com.
1. Novartis receives complete response letter from U.S. FDA for inclisiran. [press release]. December 18, 2020.
2. New Novartis analyses for investigational inclisiran demonstrate consistently effective and sustained LDL-C reduction at month 17 regardless of age and gender. [press release]. November 13, 2020.