The development of inclisiran sodium has been a relay race spanning more than a decade, combining scientific foresight and commercial courage.

The drug was originally discovered by Alnylam Pharmaceuticals, a pioneer in RNA interference (RNAi) technology, targeting the mRNA of proprotein convertase subtilisin/kexin type 9 (PCSK9).

PCSK9 protein promotes the degradation of low-density lipoprotein cholesterol (LDL-C) receptors, leading to elevated levels of “bad cholesterol” in the blood. By specifically silencing PCSK9 gene expression, inclisiran achieves sustained LDL-C reduction, offering an entirely novel mechanism for lipid management.

In 2013, Alnylam granted an exclusive license for the global development, manufacturing, and commercialization rights of inclisiran to The Medicines Company. In 2019, Novartis acquired the latter for USD 9.7 billion, gaining full control of this high-potential asset.

This bet quickly paid off through successive approvals:

• December 2020: Approved in the European Union
• December 2021: Approved by the U.S. FDA
• August 2023: Launched in the Chinese market

Today, inclisiran is available in nearly 100 countries worldwide.

For a long time, lipid management has suffered from a severe adherence crisis. For patients requiring lifelong treatment, the daily burden of statins leads approximately 50% to discontinue regular use. Although PCSK9 monoclonal antibodies offer stronger efficacy, they still require injections every 2–4 weeks.

Inclisiran’s “one injection every six months” paradigm has completely changed this reality. In patients who fail to reach LDL-C goals despite maximally tolerated statin therapy, the drug provides stable LDL-C reduction for up to 17 months.

Following an initial dose and a third-month booster, patients only require two injections per year, drastically improving treatment adherence.

Driven by its long-acting advantage, inclisiran’s sales growth has become a textbook case:

• 2021: USD 12 million
• 2022: USD 112 million (+833%)
• 2023: USD 355 million (+217%)
• 2024: USD 754 million (+112%)
• 2025: Surpassed USD 1 billion

Notably, inclisiran is not only the world’s first siRNA lipid-lowering drug but also the first small nucleic acid drug approved for a common chronic disease – a milestone achievement.

Previously, nearly all approved small nucleic acid drugs targeted rare diseases, limiting market potential. By breaking into the chronic disease space, inclisiran has shattered the market ceiling for small nucleic acid therapies.

From laboratory discovery to Novartis’s high-stakes investment, rapid global approvals, and explosive sales growth, inclisiran’s journey clearly demonstrates how cutting-edge technology can be translated into revolutionary products addressing massive unmet clinical needs.

Surpassing USD 1 billion in sales may only mark the beginning of inclisiran’s story.

In July 2023, the FDA approved an updated label allowing inclisiran to be used as an adjunct to diet and statin therapy for primary prevention in patients with elevated LDL-C and additional cardiovascular risk factors (e.g., hypertension, diabetes).

In July 2025, the FDA approved another pivotal label update: inclisiran may now be used as monotherapy alongside diet and exercise, with no mandatory requirement for combination with statins.

This represents the most transformative indication expansion since launch, positioning the RNAi therapy to shift from a “statin adjunct” to a stand-alone cornerstone treatment, expanding access to a far broader patient population.

Novartis continues to advance long-term clinical studies to unlock inclisiran’s full clinical value, expanding its role from 单纯 lipid lowering to cardiovascular event prevention – an even larger market.

Three pivotal Phase 3 trials underpin this vision:

• ORION-4 (secondary prevention): Evaluates clinical outcomes in ~15,000 patients with atherosclerotic cardiovascular disease (ASCVD). Conducted across 180 centers in the UK and US; data expected in 2026. Positive results will strongly solidify its role in secondary prevention.
• VICTORION-2P (secondary prevention): Another secondary prevention study, with key data anticipated in 2026 and 2027.
• VICTORION-1P (primary prevention): The only non-statin lipid-lowering therapy trial in high-risk primary prevention populations as defined by ACC/AHA guidelines.

Based on these clinical expansions and paradigm shifts in preventive care, China Merchants Securities (Hong Kong) forecasts peak sales of inclisiran will exceed USD 6 billion. Its ultimate market ceiling will depend directly on robust clinical evidence demonstrating benefit in cardiovascular event prevention.

In China, inclisiran sodium was first approved in August 2023 for:

• Adult patients with primary hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidemia, as an adjunct to statins;
• Or as monotherapy/combination with other non-statin lipid-lowering agents in statin-intolerant patients.

During the 2025 national medical insurance reimbursement negotiation, inclisiran was successfully included in the National Reimbursement Drug List (NRDL). Its price dropped from ¥9,988 per injection to below ¥3,000, a reduction of over 70%. With two injections annually, annual treatment cost is now under ¥6,000.

In January 2026, the NMPA approved a new indication for inclisiran: as an adjunct to diet, monotherapy for adults with primary non-familial hypercholesterolemia or mixed dyslipidemia to reduce LDL-C.

This expands coverage beyond the previously approved statin-containing regimens, reaching more patients in need.

Beyond inclisiran’s own expansion in China, its commercial success has ignited intense R&D enthusiasm among Chinese biotech firms targeting PCSK9 small nucleic acid drugs.

Beyond inclisiran’s own expansion in China, its commercial success has ignited intense R&D enthusiasm among Chinese biotech firms targeting PCSK9 small nucleic acid drugs.

Key Domestic Players in PCSK9 siRNA/Lipid-Lowering Small Nucleic Acids:

1. CSPC Pharmaceutical Group (SYH2053)Phase 3, multicenter, randomized, double-blind, placebo-controlled trial evaluating efficacy and safety as monotherapy in non-familial hypercholesterolemia and mixed dyslipidemia – patient recruitment recently initiated.

2. RiboBio & Qilu Pharmaceutical (RBD7022)Phase 2 enrollment completed in May 2025 (204 hyperlipidemia patients) to assess safety, efficacy, and long-acting lipid-lowering potential.Developed using RiboBio’s proprietary RiboGalSTARTM platform. Qilu Pharmaceutical acquired Greater China rights in December 2023 for a total deal value of ¥700 million.

3. Shengvin Pharma (SGB-3403)PCSK9 siRNA therapy backed by Eli Lilly and Roche. First patient dosed in Australia in June 2023, marking formal clinical entry.

4. Yuekang Pharmaceutical (YKYY015)SiRNA candidate for hyperlipidemia. Phase Ib initiated in November 2025 by its subsidiary.

5. Betta Pharmaceuticals / Betta-Pharma (BEBT-701)Dual-target (AGT/PCSK9) small nucleic acid drug, recently approved for Phase I–II clinical trials in patients with mild-to-moderate hypertension and elevated LDL-C.Built on Betta’s proprietary GalNAc dual oligonucleotide conjugation platform, designed to coordinately regulate blood pressure and lipid metabolism via a single agent.

Overall, as PCSK9 small nucleic acid R&D intensifies in China, competitive differentiation will hinge on:

• Further optimized dosing frequency
• Exploration of combination therapy potential
• Cost and pricing advantages from domestic manufacturing

As noted in industry interviews with Tongxieyi: as small nucleic acid drugs move into common disease markets, they compete not only with expensive innovative therapies but also with mature small-molecule generics. Crossing the significant cost divide will be decisive for commercial success.

Against the backdrop of inclisiran already being included in Chinese medical insurance, the urgency for latecomers to overcome the cost barrier and establish pricing and accessibility advantages has only grown.