TRYNGOLZA™ (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet
Ionis Pharmaceuticals (NASDAQ: IONS) announced FDA approval of TRYNGOLZA™ (olezarsen), the first-ever treatment for adults with familial chylomicronemia syndrome (FCS). The drug, administered monthly via auto-injector, demonstrated a 42.5% placebo-adjusted mean reduction in triglyceride levels at six months and 57% at 12 months in Phase 3 Balance trial. TRYNGOLZA significantly reduced acute pancreatitis events, with only one episode in the treatment group versus 11 episodes in the placebo group.
The treatment showed a favorable safety profile and will be available in the U.S. before year end. This approval marks Ionis's evolution into a commercial-stage biotechnology company, with TRYNGOLZA being the first of four planned launches over the next three years.
Ionis Pharmaceuticals (NASDAQ: IONS) ha annunciato l'approvazione della FDA per TRYNGOLZA™ (olezarsen), il primo trattamento mai sviluppato per gli adulti affetti da sindrome da chilomicronemia familiare (FCS). Il farmaco, somministrato mensilmente tramite auto-iniettore, ha mostrato una riduzione media del 42,5% dei livelli di trigliceridi, corretta per placebo, dopo sei mesi e del 57% dopo dodici mesi nello studio di Fase 3 Balance. TRYNGOLZA ha significativamente ridotto gli eventi di pancreatite acuta, con solo un episodio nel gruppo di trattamento rispetto agli 11 episodi del gruppo placebo.
Il trattamento ha dimostrato un profilo di sicurezza favorevole e sarà disponibile negli Stati Uniti entro la fine dell'anno. Questa approvazione segna l'evoluzione di Ionis in una azienda di biotecnologia nella fase commerciale, con TRYNGOLZA che rappresenta il primo di quattro lanci previsti nei prossimi tre anni.
Ionis Pharmaceuticals (NASDAQ: IONS) anunció la aprobación de la FDA para TRYNGOLZA™ (olezarsen), el primer tratamiento desarrollado jamás para adultos con síndrome de quilomicronemia familiar (FCS). El fármaco, administrado mensualmente mediante un autoinyector, mostró una reducción media del 42,5% en los niveles de triglicéridos ajustada por placebo a los seis meses y del 57% a los doce meses en el ensayo de Fase 3 Balance. TRYNGOLZA redujo significativamente los episodios de pancreatitis aguda, con solo un episodio en el grupo de tratamiento frente a 11 episodios en el grupo placebo.
El tratamiento mostró un perfil de seguridad favorable y estará disponible en EE. UU. antes de fin de año. Esta aprobación marca la evolución de Ionis hacia una empresa de biotecnología en etapa comercial, con TRYNGOLZA siendo el primero de cuatro lanzamientos programados durante los próximos tres años.
아이오니스 제약 (NASDAQ: IONS)은 TRYNGOLZA™(올레자르센)의 FDA 승인을 발표했습니다. 이는 가족성 킬로미크론 혈증 증후군(FCS)을 가진 성인을 위한 최초의 치료제입니다. 이 약물은 매달 자가 주사기로 투여되며, 3상 밸런스 시험에서 6개월 후에 플라시보 조정 의미에서 42.5%의 중성지방 수치 감소를 보여주었고, 12개월 후에는 57%의 감소를 보였습니다. TRYNGOLZA는 급성 췌장염 사건을 유의미하게 줄였으며, 치료군에서 발생한 사건은 단 한 건이었고, 플라시보 군에서는 11건이 발생했습니다.
이 치료제는 유리한 안전 프로파일을 보여주었으며, 연말 전에 미국에서 제공될 예정입니다. 이 승인은 아이오니스가 상업 단계의 생명공학 기업으로 발전해가는 것을 의미하며, TRYNGOLZA는 향후 3년 간 4개의 출시 중 첫 번째가 될 것입니다.
Ionis Pharmaceuticals (NASDAQ: IONS) a annoncé l'approbation de la FDA pour TRYNGOLZA™ (olezarsen), le premier traitement jamais proposé aux adultes atteints du syndrome de chylomicronémie familiale (FCS). Le médicament, administré une fois par mois via un auto-injecteur, a montré une réduction moyenne des niveaux de triglycérides ajustée par placebo de 42,5 % après six mois et de 57 % après douze mois lors de l'essai clinique de Phase 3 Balance. TRYNGOLZA a considérablement réduit les événements de pancréatite aiguë, avec un seul épisode dans le groupe traité contre 11 épisodes dans le groupe placebo.
Le traitement a présenté un profil de sécurité favorable et sera disponible aux États-Unis avant la fin de l'année. Cette approbation marque l'évolution d'Ionis vers une entreprise de biotechnologie à stade commercial, avec TRYNGOLZA comme premier d'une série de quatre lancements prévus au cours des trois prochaines années.
Ionis Pharmaceuticals (NASDAQ: IONS) gab die FDA-Zulassung für TRYNGOLZA™ (olezarsen) bekannt, die erste Behandlung für Erwachsene mit familiärem Chylomikronämiesyndrom (FCS). Das Medikament, das monatlich über einen Autoinjektor verabreicht wird, zeigte eine placebo-adjustierte mittlere Reduktion der Triglyceridwerte von 42,5 % nach sechs Monaten und 57 % nach zwölf Monaten in der Phase-3-Balance-Studie. TRYNGOLZA verringerte signifikant die Fälle von akuter Pankreatitis, mit nur einem Vorfall in der Behandlungsgruppe im Vergleich zu 11 Vorfällen in der Placebogruppe.
Die Behandlung zeigte ein günstiges Sicherheitsprofil und wird noch vor Jahresende in den USA verfügbar sein. Diese Zulassung markiert die Entwicklung von Ionis zu einem biopharmazeutischen Unternehmen in der kommerziellen Phase, wobei TRYNGOLZA das erste von vier geplanten Markteinführungen in den nächsten drei Jahren darstellt.
- First-ever FDA-approved treatment for FCS
- Significant 57% reduction in triglycerides at 12 months
- Substantial reduction in acute pancreatitis events (1 vs 11 episodes)
- First of four planned product launches in next three years
- Priority Review, Fast Track, Orphan Drug, and Breakthrough Therapy designations received
- Common adverse reactions including injection site reactions (19%)
- Decreased platelet count reported in 12% of treated patients
- market size (approximately 3,000 people in U.S.)
Insights
The FDA approval of TRYNGOLZA marks a groundbreaking milestone in rare disease treatment. The drug demonstrated remarkable efficacy with a
The market opportunity, while focused with approximately 3,000 U.S. patients, could be significant given TRYNGOLZA's unique position as the first approved therapy. With monthly administration and strong efficacy data published in NEJM, this approval strengthens Ionis' commercial portfolio and validates their antisense technology platform. The Priority Review, Fast Track, Orphan Drug and Breakthrough Therapy designations underscore the drug's importance.
This launch initiates Ionis' strategic transformation into a commercial-stage company, with three more potential launches planned in the next three years. The robust safety profile and comprehensive patient support program should facilitate market adoption.
This approval represents a significant commercial opportunity for Ionis. With no existing treatments for FCS, TRYNGOLZA enters an untapped market as a first-in-class therapy. The rare disease pricing model typically commands premium pricing and with 3,000 potential U.S. patients, the revenue potential could be substantial.
The company's transition to a commercial entity with its own launches marks a strategic evolution that could positively impact valuation metrics. The planned launch of four independent products over three years suggests a robust near-term growth trajectory. The comprehensive Ionis Every Step™ support program demonstrates commercial readiness and should help overcome access barriers typical in rare disease markets.
The clinical impact of TRYNGOLZA is remarkable from a medical perspective. The reduction in acute pancreatitis events - from 11 episodes in 7 placebo patients to just 1 episode in 1 treated patient - represents a paradigm shift in FCS management. The sustained triglyceride reductions over 12 months suggest durable efficacy.
The favorable safety profile, with mainly manageable injection site reactions and modest platelet count decreases, supports long-term use. The monthly self-administered dosing via auto-injector should enhance treatment adherence compared to daily oral medications. The indication's breadth, covering both genetically and clinically confirmed FCS, maximizes the accessible patient population.
- TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease
- Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis
- TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals
- Ionis to host webcast today at 6:45pm ET
Experience the full interactive Multichannel News Release here: https://www.multivu.com/ionis-pharmaceuticals/9295551-en-tryngolza-olezarsen-fda-approval
"Today's FDA approval of TRYNGOLZA heralds the arrival of the first-ever FCS treatment in the
The FDA approval was based on positive data from the global, multicenter, randomized, placebo-controlled, double-blind Phase 3 Balance clinical trial in adult patients with genetically identified FCS and fasting triglyceride levels ≥880 mg/dL. In the Balance study, TRYNGOLZA 80 mg demonstrated a statistically significant placebo-adjusted mean reduction in triglyceride levels of
TRYNGOLZA demonstrated a favorable safety profile. The most common adverse reactions (incidence >
Results from the Phase 3 Balance study were previously published in The New England Journal of Medicine (NEJM).
"With no treatment options previously available, we were limited to relying only on extremely strict diet and lifestyle changes as the sole preventative treatment option," said Alan Brown, M.D., FNLA, FACC, FAHA, clinical professor of medicine, Rosalind Franklin University of Medicine and Science; Balance trial investigator. "The FDA approval of TRYNGOLZA is an important moment for people living with FCS, their families and physicians who now, for the first time, have a treatment that significantly lowers triglycerides and decreases the risk of potentially life-threatening acute pancreatitis events, as an adjunct to a low-fat diet. I am excited to have a medicine I can prescribe to my patients that has been shown to change the course of their disease."
FCS is a rare, genetic, potentially life-threatening form of sHTG that prevents the body from breaking down fats and severely impairs the body's ability to remove triglycerides from the bloodstream due to an impaired function of the enzyme lipoprotein lipase (LPL). While healthy levels for adults are below 150 mg/dL, people with FCS often have triglyceride levels of more than 880 mg/dL and often have a history of pancreatitis. Those living with FCS have a high risk of potentially fatal AP, which is a painful inflammation of the pancreas, and chronic health issues such as fatigue and severe, recurrent abdominal pain. People living with FCS can also experience psychological and financial stress, which can significantly impact their quality of life. In the
"As a rare and difficult to diagnose disease, FCS has a profound impact on the lives of patients and families. Many people living with FCS have experienced severe pain their whole lives – sometimes so intense they require lengthy hospitalization stays – and struggle through life with daily fatigue, nausea, brain fog and stomach pain," said Lindsey Sutton Bryan, co-founder and co-president, FCS Foundation. "Until now, our treatment options have been limited, relying on diet alone to try to manage triglyceride levels and keep acute pancreatitis attacks at bay. For the first time, adults with FCS have seen their hope for a treatment become a reality."
TRYNGOLZA will be available in the
Ionis is committed to helping people access the medicines they are prescribed and will offer a suite of services designed to meet the unique needs of the FCS community through Ionis Every Step™. As part of Ionis Every Step, patients and healthcare providers will have access to services throughout the treatment journey provided by dedicated Patient Education Managers and Ionis Every Step Case Managers, including insurance and affordability support, as well as services and resources, such as disease and nutrition education. Visit TRYNGOLZA.com for more information.
TRYNGOLZA was reviewed by the FDA under Priority Review and had previously been granted Fast Track designation for the treatment of FCS, Orphan Drug designation and Breakthrough Therapy designation. Olezarsen is undergoing review in the European Union and regulatory filings in other countries are planned. Olezarsen is currently being evaluated in three Phase 3 clinical trials – CORE, CORE2 and ESSENCE – for the treatment of sHTG. Olezarsen has not been reviewed or approved for the treatment of sHTG by regulatory authorities.
Webcast
Ionis will hold a webcast today at 6:45pm ET to discuss the FDA approval. Interested parties may access the webcast here. A webcast replay will be available for a limited time.
About TRYNGOLZA™ (olezarsen)
TRYNGOLZA™ (olezarsen) was approved by the
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence >
Please see full Prescribing Information for TRYNGOLZA.
About Familial Chylomicronemia Syndrome (FCS)
FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are
About the Balance Study
Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters and adjudicated acute pancreatitis event rates over the treatment period. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every four weeks.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has discovered and developed six marketed medicines for serious diseases, including breakthrough medicines for neurologic and cardiovascular diseases. Ionis has a leading pipeline in neurology, cardiology and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.
Ionis Forward-Looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of TRYNGOLZA, Ionis' technologies and other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals® and TRYNGOLZA™ are trademarks of Ionis Pharmaceuticals, Inc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
info@ionis.com 760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.
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