Overview
Inclisiran (The Medicines Company/Alnylam Pharmaceuticals) is a systemically delivered siRNA therapeutic targeting mRNA for PCSK. Inclisiran targets both intracellular and extracellular PCSK9, thereby producing the same effect observed in loss of function or null human PCSK9 mutations, without any adverse effects on high-density lipoprotein cholesterol levels.
Inclisiran is a small interfering ribonucleic acid (siRNA) product, engineered to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) production. The product is expected to have comparable efficacy to monoclonal antibody (MAb) PCSK9 inhibitors, Repatha (evolocumab; Amgen) and Praluent (alirocumab; Sanofi/Regeneron). However, inclisiran may be able to differentiate from the MAb PCSK9 products by offering patients an advantageous dosing schedule at a discounted cost to the referenced products. It is likely that patients will only be required to take inclisiran two or three times a year, while the MAb PCSK9s are required to be dosed at a minimum of once a month. By not requiring as frequent doses, a course of treatment with inclisiran may be priced at a discount to MAb PCSK9s. The expected lower cost of inclisiran may make it easier for The Medicines Company and Alnylam to negotiate broader reimbursement with insurers, something which has inhibited growth of the MAb products. The Medicines Company initiated its Phase III trial program for inclisiran in Q4 2017, and hopes to file a New Drug Application with the US Food and Drug Administration in 2019.
Inclisiran (The Medicines Company/Alnylam Pharmaceuticals) is a systemically delivered siRNA therapeutic targeting mRNA for PCSK. Inclisiran targets both intracellular and extracellular PCSK9, thereby producing the same effect observed in loss of function or null human PCSK9 mutations, without any adverse effects on high-density lipoprotein cholesterol levels.
Inclisiran is a small interfering ribonucleic acid (siRNA) product, engineered to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) production. The product is expected to have comparable efficacy to monoclonal antibody (MAb) PCSK9 inhibitors, Repatha (evolocumab; Amgen) and Praluent (alirocumab; Sanofi/Regeneron). However, inclisiran may be able to differentiate from the MAb PCSK9 products by offering patients an advantageous dosing schedule at a discounted cost to the referenced products. It is likely that patients will only be required to take inclisiran two or three times a year, while the MAb PCSK9s are required to be dosed at a minimum of once a month. By not requiring as frequent doses, a course of treatment with inclisiran may be priced at a discount to MAb PCSK9s. The expected lower cost of inclisiran may make it easier for The Medicines Company and Alnylam to negotiate broader reimbursement with insurers, something which has inhibited growth of the MAb products. The Medicines Company initiated its Phase III trial program for inclisiran in Q4 2017, and hopes to file a New Drug Application with the US Food and Drug Administration in 2019.
Table of Contents
OVERVIEW
LIST OF FIGURES
LIST OF TABLES
