Inclisiran: A Game-Changer in Cardiovascular Disease Management

The RNAi therapeutics market is undergoing a seismic shift, driven by groundbreaking advancements in gene-silencing technologies and their application to chronic diseases like cardiovascular disorders. At the forefront of this revolution is Novartis’ Leqvio (inclisiran), a small interfering RNA (siRNA) therapy that has redefined the management of hypercholesterolemia. With a projected CAGR of 14.9% for the global RNAi therapeutics market from 2025 to 2034 [1], and cardiovascular applications accounting for a significant portion of this growth, inclisiran’s strategic positioning and clinical efficacy make it a compelling long-term investment.

The Science and Strategic Edge of Inclisiran

Inclisiran’s mechanism of action—targeting the PCSK9 gene to reduce low-density lipoprotein cholesterol (LDL-C)—offers a paradigm shift in lipid management. Clinical trials, such as the V-DIFFERENCE study, demonstrate that 85% of patients on Leqvio plus optimized lipid-lowering therapy achieved LDL-C targets within 90 days, compared to 31% on placebo [2]. This efficacy, combined with a biannual dosing regimen, addresses critical gaps in patient adherence and cost efficiency. For context, competitors like Amgen’s Repatha require monthly infusions, creating a logistical and financial burden that inclisiran’s twice-yearly administration avoids [3].

Novartis has further strengthened its position by securing FDA approval for Leqvio as a first-line monotherapy in 2025, eliminating the prior requirement for concurrent statin use [4]. This expansion broadens the drug’s addressable market to include up to 80% of atherosclerotic cardiovascular disease (ASCVD) patients who struggle to meet LDL-C targets [4]. The approval aligns with evolving clinical guidelines emphasizing aggressive LDL-C reduction, positioning Leqvio as a flexible and patient-centric solution.

Market Dynamics and Competitive Landscape

The hypercholesterolemia market is poised for exponential growth, fueled by the rising prevalence of cardiometabolic disorders and the introduction of RNAi-based therapies. Inclisiran’s siRNA modality dominates the RNAi therapeutics segment, holding a 64% market share in 2023 [5]. With a projected CAGR of 35.4% from 2025 to 2032 for the RNAi therapeutics market [5], Novartis is capitalizing on a high-margin, innovation-driven sector.

Financial metrics underscore inclisiran’s potential: Leqvio is forecasted to reach $1.2 billion in sales in 2025, with a 15-20% market share by 2027 [3]. This growth is further supported by Novartis’ recent agreement with the pan-Canadian Pharmaceutical Alliance (pCPA) for public reimbursement in Canada, addressing a key barrier to adoption for patients with heterozygous familial hypercholesterolemia (HeFH) [4]. While broader access initiatives remain pending, the drug’s unique value proposition—proven efficacy, reduced adverse events, and cost efficiency—positions it to outpace competitors in the PCSK9 inhibitors market, which is expected to grow at a 21.2% CAGR through 2032 [6].

Long-Term Investment Considerations

Investors should consider the broader implications of RNAi technology in cardiovascular care. The ability to silence disease-causing genes with high specificity and minimal off-target effects [5] opens avenues for treating conditions beyond hypercholesterolemia, such as hypertension and type 2 diabetes. Alnylam Pharmaceuticals, a key partner in inclisiran’s development, has demonstrated leadership in RNAi innovation, with therapies like AMVUTTRA (vutrisiran) showing promise in ATTR amyloidosis [7]. This ecosystem of innovation reinforces the durability of Novartis’ investment in RNAi.

However, challenges remain. Long-term cardiovascular outcome data for inclisiran is still limited, with only 11 of 92 clinical trials as of late 2024 focused on cardiovascular endpoints [8]. Real-world evidence on adherence and cost-effectiveness will be critical for sustained market penetration. Nonetheless, the drug’s early success and Novartis’ aggressive expansion strategy suggest a strong trajectory.

Conclusion

Inclisiran represents a transformative force in cardiovascular disease management, leveraging RNAi’s precision and durability to address unmet medical needs. Novartis’ strategic approvals, dosing innovation, and access initiatives position Leqvio as a cornerstone of lipid-lowering therapy. For investors, the convergence of a high-growth market, a robust clinical profile, and a first-mover advantage in RNAi therapeutics makes inclisiran a compelling long-term bet.

Source:
[1] RNAi Therapeutics Market Size Leads 14.9% CAGR by 2034 [https://www.towardshealthcare.com/insights/rnai-therapeutics-market-sizing]
[2] Novartis Leqvio® shows statistically significant and clinically meaningful early LDL-C goal achievement with less muscle pain [https://www.novartis.com/news/media-releases/novartis-leqvio-shows-statistically-significant-and-clinically-meaningful-early-ldl-c-goal-achievement-less-muscle-pain]
[3] The Investment Case for Novartis' Leqvio in the Evolving Cardiovascular Therapeutics Market [https://www.ainvest.com/news/investment-case-novartis-leqvio-evolving-cardiovascular-therapeutics-market-2508/]
[4] Novartis twice-yearly* Leqvio® (inclisiran) receives FDA approval new indication enabling first-line use [https://www.novartis.com/us-en/news/media-releases/novartis-twice-yearly-leqvio-inclisiran-receives-fda-approval-new-indication-enabling-first-line-use]
[5] RNAi Therapeutics Market Trends and Top Players by 2031 [https://www.theinsightpartners.com/reports/rnai-therapeutics-market]
[6] PCSK9 Inhibitors Market Report | Global Forecast From [https://dataintelo.com/report/global-pcsk9-inhibitors-market]
[7] Alnylam Pharmaceuticals Reports First Quarter 2025 Financial [https://investors.alnylam.com/press-release?id=28956]
[8] Current usage of inclisiran for cardiovascular diseases [https://pmc.ncbi.nlm.nih.gov/articles/PMC11868277/]