Drug Overview
Kynamro (mipomersen sodium) was the first antisense therapy to enter the dyslipidemia market. It is marketed by Kastle Therapeutics and is approved in the US for the treatment of homozygous familial hypercholesterolemia (HoFH). The molecule is a 20-base, second-generation antisense oligonucleotide (ASO), which forms a sequence-specific duplex with the messenger RNA (mRNA) for apolipoprotein B-100 (ApoB-100). The duplex then serves as a substrate for ribonuclease H, which cleaves the ASO-bound mRNA, ultimately leading to reduced translation of ApoB-100. ApoB-100 is the primary structural protein of atherogenic lipoproteins, including low-density lipoprotein (LDL). Therefore, by reducing ApoB-100 concentrations, Kynamro also reduces plasma LDL cholesterol concentrations.
Kynamro has inferior efficacy and tolerability, and an inferior administration route compared to competing HoFH therapy, Juxtapid (lomitapide mesylate; Aegerion/Amryt). Kynamro has experienced poor uptake since its launch due to its inability to compete with Juxtapid, despite its lower price. Kynamro is facing further competition from the market entry of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors Repatha (evolocumab; Amgen/Astellas) and Praluent (alirocumab; Sanofi/Regeneron), which provide a safer, cheaper, and more efficacious alternative. Future revenues are not expected to increase as market and label expansions appear unlikely due to ongoing safety concerns.
Kynamro (mipomersen sodium) was the first antisense therapy to enter the dyslipidemia market. It is marketed by Kastle Therapeutics and is approved in the US for the treatment of homozygous familial hypercholesterolemia (HoFH). The molecule is a 20-base, second-generation antisense oligonucleotide (ASO), which forms a sequence-specific duplex with the messenger RNA (mRNA) for apolipoprotein B-100 (ApoB-100). The duplex then serves as a substrate for ribonuclease H, which cleaves the ASO-bound mRNA, ultimately leading to reduced translation of ApoB-100. ApoB-100 is the primary structural protein of atherogenic lipoproteins, including low-density lipoprotein (LDL). Therefore, by reducing ApoB-100 concentrations, Kynamro also reduces plasma LDL cholesterol concentrations.
Kynamro has inferior efficacy and tolerability, and an inferior administration route compared to competing HoFH therapy, Juxtapid (lomitapide mesylate; Aegerion/Amryt). Kynamro has experienced poor uptake since its launch due to its inability to compete with Juxtapid, despite its lower price. Kynamro is facing further competition from the market entry of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors Repatha (evolocumab; Amgen/Astellas) and Praluent (alirocumab; Sanofi/Regeneron), which provide a safer, cheaper, and more efficacious alternative. Future revenues are not expected to increase as market and label expansions appear unlikely due to ongoing safety concerns.
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