Novartis twice-yearly* Leqvio® (inclisiran) receives FDA approval for new indication enabling first-line use
Novartis (NYSE:NVS) announced a significant FDA approval for a label update of Leqvio® (inclisiran), enabling its use as a first-line monotherapy for LDL-C reduction in adults with hypercholesterolemia. The update removes the requirement for combination with statin therapy, allowing Leqvio to be used alone with diet and exercise.
The FDA proactively requested this label update based on robust LDL-C lowering data for PCSK9-targeting therapies. This development is particularly significant as up to 80% of ASCVD patients in the US struggle to reach the LDL-C guideline-recommended target of <70 mg/dL. Leqvio's unique twice-yearly dosing regimen, administered by healthcare providers, aims to support better patient adherence and long-term cholesterol management.
Novartis (NYSE:NVS) ha annunciato un'importante approvazione della FDA per un aggiornamento dell'etichetta di Leqvio® (inclisiran), che ne consente l'uso come monoterapia di prima linea per la riduzione del LDL-C negli adulti con ipercolesterolemia. L'aggiornamento elimina la necessità di combinare il farmaco con la terapia a base di statine, permettendo l'uso di Leqvio da solo insieme a dieta ed esercizio fisico.
La FDA ha richiesto proattivamente questo aggiornamento dell'etichetta basandosi su dati solidi riguardanti la riduzione del LDL-C con terapie che mirano a PCSK9. Questo sviluppo è particolarmente rilevante poiché fino all'80% dei pazienti con ASCVD negli Stati Uniti fatica a raggiungere il target raccomandato dalle linee guida per il LDL-C di <70 mg/dL. Il regime unico di somministrazione di Leqvio, due volte all'anno e effettuato da operatori sanitari, mira a migliorare l'aderenza dei pazienti e la gestione a lungo termine del colesterolo.
Novartis (NYSE:NVS) anunció una importante aprobación de la FDA para una actualización de la etiqueta de Leqvio® (inclisiran), que permite su uso como monoterapia de primera línea para la reducción del LDL-C en adultos con hipercolesterolemia. La actualización elimina el requisito de combinarlo con terapia con estatinas, permitiendo que Leqvio se use solo junto con dieta y ejercicio.
La FDA solicitó proactivamente esta actualización de la etiqueta basándose en datos sólidos sobre la reducción del LDL-C con terapias dirigidas a PCSK9. Este avance es especialmente significativo ya que hasta el 80% de los pacientes con ASCVD en EE.UU. tienen dificultades para alcanzar el objetivo de LDL-C recomendado por las guías de <70 mg/dL. El régimen único de dosificación de Leqvio, administrado dos veces al año por profesionales de la salud, busca mejorar la adherencia del paciente y el manejo a largo plazo del colesterol.
노바티스(NYSE:NVS)는 Leqvio® (인클리시란)의 라벨 업데이트에 대한 중요한 FDA 승인을 발표했습니다. 이로써 고콜레스테롤혈증 성인의 LDL-C 감소를 위한 1차 단독 요법으로 사용이 가능해졌습니다. 이번 업데이트로 스타틴 병용 요법 요구 사항이 제거되어 Leqvio를 식이요법 및 운동과 함께 단독으로 사용할 수 있게 되었습니다.
FDA는 PCSK9 표적 치료제의 강력한 LDL-C 감소 데이터를 바탕으로 이 라벨 업데이트를 선제적으로 요청했습니다. 이 발전은 미국 내 ASCVD 환자의 최대 80%가 LDL-C 가이드라인 권장 목표인 <70 mg/dL에 도달하지 못하는 상황에서 특히 중요합니다. Leqvio의 독특한 연 2회 투여 방식은 의료 제공자가 투여하며, 환자의 복약 순응도와 장기적인 콜레스테롤 관리를 지원하는 것을 목표로 합니다.
Novartis (NYSE:NVS) a annoncé une approbation importante de la FDA concernant une mise à jour de l'étiquette de Leqvio® (inclisiran), permettant son utilisation en monothérapie de première intention pour la réduction du LDL-C chez les adultes atteints d'hypercholestérolémie. Cette mise à jour supprime l'obligation d'associer le traitement aux statines, autorisant ainsi l'utilisation de Leqvio seul, en complément d'un régime alimentaire et d'exercice physique.
La FDA a demandé proactivement cette mise à jour d'étiquette sur la base de données solides démontrant la réduction du LDL-C avec les thérapies ciblant PCSK9. Ce développement est particulièrement significatif car jusqu'à 80 % des patients atteints de maladie cardiovasculaire athéroscléreuse (ASCVD) aux États-Unis ont du mal à atteindre l'objectif recommandé de LDL-C inférieur à <70 mg/dL. Le schéma posologique unique de Leqvio, administré deux fois par an par des professionnels de santé, vise à améliorer l'adhésion des patients et la gestion à long terme du cholestérol.
Novartis (NYSE:NVS) gab eine bedeutende FDA-Zulassung für ein Label-Update von Leqvio® (Inclisiran) bekannt, das die Anwendung als Erstlinientherapie zur LDL-C-Senkung bei Erwachsenen mit Hypercholesterinämie ermöglicht. Das Update hebt die Notwendigkeit der Kombination mit Statintherapie auf, sodass Leqvio allein zusammen mit Diät und Bewegung eingesetzt werden kann.
Die FDA forderte dieses Label-Update proaktiv aufgrund robuster Daten zur LDL-C-Senkung mit PCSK9-zielgerichteten Therapien an. Diese Entwicklung ist besonders bedeutsam, da bis zu 80 % der ASCVD-Patienten in den USA Schwierigkeiten haben, den LDL-C-Zielwert von <70 mg/dL gemäß Leitlinien zu erreichen. Das einzigartige halbjährliche Dosierungsschema von Leqvio, das von medizinischem Personal verabreicht wird, soll die Patienten-Compliance und das langfristige Cholesterinmanagement verbessern.
- FDA approval for first-line monotherapy use expands Leqvio's market potential
- Twice-yearly dosing schedule supports better patient adherence
- FDA proactively requested the label update, demonstrating confidence in the treatment
- Addresses large market opportunity with 80% of ASCVD patients not reaching LDL-C targets
- Still requires healthcare provider administration, limiting accessibility compared to self-administered alternatives
Insights
FDA approval for Leqvio as monotherapy significantly expands Novartis's addressable market in high-cholesterol patients, boosting commercial potential.
This FDA label update for Leqvio represents a significant commercial opportunity for Novartis by removing a key limitation in the drug's prescription pathway. Previously, patients had to use Leqvio in combination with statins, creating a narrower target population. Now, as a first-line monotherapy option, Novartis can target the broader hypercholesterolemia market directly, including statin-intolerant patients and those preferring to avoid daily medication.
The timing aligns perfectly with the 2025 ACC/AHA guidelines recommending more aggressive LDL-C targets. With 80% of ASCVD patients failing to reach recommended LDL-C levels, there's substantial unmet need that Leqvio can now address earlier in the treatment journey. The twice-yearly administration schedule gives Leqvio a compelling adherence advantage over daily oral medications or even monthly/bi-monthly injectable PCSK9 inhibitors.
Particularly noteworthy is that the FDA proactively requested this label update, indicating regulatory confidence in Leqvio's efficacy and safety profile as monotherapy. This removes significant regulatory risk that would normally accompany label expansion applications. For Novartis, this approval strengthens their cardiovascular portfolio at a time when many pharmaceutical companies face patent cliffs and pricing pressures in established therapeutic areas. The expanded Leqvio indication positions Novartis to capture greater market share in the hypercholesterolemia space with minimal additional investment in clinical trials.
- Leqvio can now be used alone, without addition of statin therapy, as adjunct to diet and exercise for LDL-C reduction in patients with hypercholesterolemia (high LDL-C)1
- 4 out of 5 atherosclerotic cardiovascular disease (ASCVD) patients do not reach guideline-recommended LDL-C target, reinforcing urgent need for more aggressive LDL-C lowering2-5
- Twice-yearly Leqvio is uniquely positioned to help support patient adherence and long-term LDL-C management, including goal attainment
"This first-line label update reinforces Leqvio's proven ability to effectively lower LDL-C, a critical risk factor for heart disease," said Victor Bultó, President, US, Novartis. "With this new indication enabling Leqvio's use as monotherapy along with diet and exercise, we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey."
With its twice-yearly, health care provider-administered dosing, Leqvio is uniquely positioned to help support patient adherence and long-term LDL-C management, including goal attainment. This is a critical unmet need, as up to
The updated label removes the requirement for Leqvio to be used on top of or in combination with statin therapy1. Other updates include revising "primary hyperlipidemia" to the more specific term of "hypercholesterolemia" throughout the label, to more accurately focus on LDL-C reduction1.
*After an initial dose and another at three months.
About Leqvio
Leqvio is an injectable prescription medicine indicated as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH).
Novartis has obtained global rights to develop, manufacture and commercialize Leqvio under a license and collaboration agreement with Alnylam Pharmaceuticals, a leader in RNAi therapeutics.
Important Safety Information
Do not use if you have had an allergic reaction to Leqvio or any of its ingredients. The most common side effects of Leqvio were injection site reaction (including pain, redness, and rash), arthralgia (joint pain), bronchitis (chest cold).
These are not all the possible side effects of Leqvio. Ask your health care provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please click here for Leqvio full Prescribing Information.
About Atherosclerotic Cardiovascular Disease (ASCVD)
Cardiovascular disease (CVD) affects hundreds of millions of people and claims more lives globally than cancer, chronic lung disease and diabetes combined7. Around
ASCVD accounts for
About Novartis in Cardiovascular Disease
At Novartis, our mission is to ensure no heart is lost too soon. We envision a world where preventable CV deaths are no longer part of our lives. We're proud of the positive impact we've made over the past 40 years and remain dedicated to tackling the most challenging problems in CVD. Through cutting-edge science and technology, we are focusing on areas of high unmet need, including scaling our xRNA platform across multiple risk factors and pioneering breakthroughs for genetically driven CVD risk factors and common heart conditions, including atrial fibrillation.
We also work with patients, healthcare professionals, and organizations around the world to improve CV care beyond medicine alone. Together, we can help people with CVD enjoy longer, healthier lives and more time with their loved ones.
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.
Reimagine medicine with us: Visit us at https://www.novartis.us and connect with us on LinkedIn US, X/Twitter US and Instagram.
References
- Leqvio. Prescribing information. Novartis Pharmaceuticals Corp
- Cannon CP, de Lemos JA, Rosenson RS, et al. Use of lipid-lowering therapies over 2 years in GOULD, a registry of patients with atherosclerotic cardiovascular disease in the US. JAMA Cardiol. 2021;6(9):1060-1068. doi:10.1001/jamacardio.2021.1810
- Fox KM, Tai M-H, Kostev K, Hatz M, Qian Y, Laufs U. Treatment patterns and low-density lipoprotein cholesterol (LDL-C) goal attainment among patients receiving high- or moderate-intensity statins. Clin Res Cardiol. 2018;107(5):380-388. doi:10.1007/s00392-017-1193-z
- Wong ND, Young D, Zhao Y, et al. Prevalence of the American College of Cardiology/American Heart Association statin eligibility groups, statin use, and low-density lipoprotein cholesterol control in US adults using the National Health and Nutrition Examination Survey 2011-2012. J Clin Lipidol. 2016;10(5):1109-1118. doi:10.1016/j.jacl.2016.06.011
- Grundy SM, Stone NJ, Bailey AL, et al. 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA guideline on the management of blood cholesterol: a report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines. Circulation. 2019;139(25):e1082-e1143. doi:10.1016/j.jacc.2018.11.002
- Rao SV, O'Donoghue ML, Ruel M, et al. 2025 ACC/AHA/ACEP/NAEMSP/SCAI guideline for the management of patients with acute coronary syndromes: a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. J Am Coll Cardiol. 2025;85(22):2135-2237. doi:10.1016/j.jacc.2024.11.009
- American Heart Association. More than half of
U.S. adults don't know heart disease is leading cause of death, despite 100-year reign. Published January 24, 2024. Accessed July 25, 2025. https://newsroom.heart.org/news/more-than-half-of-u-s-adults-dont-know-heart-disease-is-leading-cause-of-death-despite-100-year-reign - World Heart Federation. World Heart Report 2023: confronting the world's number one killer. Published May 20, 2023. Accessed July 25, 2025. https://world-heart-federation.org/wp-content/uploads/World-Heart-Report-2023.pdf
- World Health Organization. Cardiovascular diseases (CVDs). Published June 11, 2021. Accessed July 25, 2025. https://www.who.int/news-room/fact-sheets/detail/cardiovascular-diseases-(cvds)
- Roger VL, Go AS, Lloyd-Jones DM, et al. Heart disease and stroke statistics–2012 update: a report from the American Heart Association. Circulation. 2012;125(1):e2-e220. doi:10.1161/CIR.0b013e31823ac046
- Kim H, Kim S, Han S, et al. Prevalence and incidence of atherosclerotic cardiovascular disease and its risk factors in
Korea : a nationwide population-based study. BMC Public Health. 2019;19(1):1112. doi:10.1186/s12889-019-7439-0 - Goldstein JL, Brown MS. A century of cholesterol and coronaries: from plaques to genes to statins. Cell. 2015;161(1):161-172. doi:10.1016/j.cell.2015.01.036
- Ference BA, Graham I, Tokgozoglu L, Catapano AL. Impact of lipids on cardiovascular health: JACC Health Promotion Series. J Am Coll Cardiol. 2018;72(10):1141-1156. doi:10.1016/j.jacc.2018.06.046
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FAQ
What is the new FDA approval for Novartis Leqvio (NVS)?
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