The Scottish Medicines Consortium (SMC) has delivered a significant setback to Daiichi Sankyo's cholesterol-lowering ambitions by rejecting Nilemdo (bempedoic acid) for use within NHS Scotland, citing inadequate evidence to support the drug's adoption.
After reviewing data from four phase 3 clinical trials, the SMC concluded that Daiichi Sankyo failed to present sufficiently robust clinical and economic analyses to warrant acceptance. This decision comes as the healthcare community awaits NICE's verdict for England and Wales.
Clinical Profile and Regulatory Status
Nilemdo, developed by US-based Esperion and licensed to Daiichi Sankyo in Europe, received EMA approval in April following FDA authorization. The drug is indicated for patients with abnormally high cholesterol or established atherosclerotic cardiovascular disease who either cannot tolerate statins or remain on maximum-tolerated statin therapy, with the option for combination treatment with ezetimibe.
The once-daily oral medication launched in Germany last month, with UK market entry planned for early 2023. This novel therapeutic approach offers an alternative for patients struggling with statin therapy, addressing an important gap in cardiovascular disease management.
Market Implications and Competition
Industry analysts had projected peak annual sales of $3 billion for Nilemdo/Nexletol, though this forecast may require revision if other health technology assessment bodies follow SMC's position. The Institute for Clinical and Economic Review (ICER) in the US has already expressed concerns about the drug's cost-effectiveness in a draft report.
The cholesterol-lowering market has proven challenging, as evidenced by the underwhelming performance of injectable PCSK9 inhibitors like Amgen's Repatha and Sanofi/Regeneron's Praluent. While Nilemdo offers the advantage of oral administration, it faces potential competition from Novartis's Leqvio (inclisiran), a twice-yearly injectable PCSK9 inhibitor awaiting regulatory decisions.
Commercial Strategy and Future Outlook
Despite this setback, Daiichi Sankyo has demonstrated continued commitment to the European market, recently securing full European rights to both Nilemdo and its combination product Nustendi through a $150 million milestone payment. The company now faces the challenge of convincing other European health technology assessment agencies of Nilemdo's value proposition.
The outcome of pending NICE evaluation and other European regulatory decisions will be crucial in determining Nilemdo's commercial trajectory and its potential to address the ongoing challenges in managing cardiovascular disease.
