BEYONTTRA® (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved by the UK Medicines and Healthcare Products Regulatory Agency to Treat ATTR-CM
BridgeBio Pharma (BBIO) has received UK approval for BEYONTTRA® (acoramidis), the first near-complete TTR stabilizer (≥90%) for treating ATTR-CM (transthyretin amyloidosis with cardiomyopathy). The approval is based on the Phase 3 ATTRibute-CM study results, which demonstrated:
- 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events at Month 30
- 50% reduction in cumulative frequency of cardiovascular hospitalization events
- Rapid benefit showing separation from placebo in as few as 3 months
BEYONTTRA is now approved in the US, EU, UK, and Japan. Under a collaboration agreement with Bayer, who will handle UK commercialization, BridgeBio will receive tiered royalties starting in the low-thirties percent on sales.
BridgeBio Pharma (BBIO) ha ottenuto l'approvazione nel Regno Unito per BEYONTTRA® (acoramidis), il primo stabilizzatore di TTR quasi completo (≥90%) per il trattamento dell'ATTR-CM (amiloidosi da transtiretina con cardiomiopatia). L'approvazione si basa sui risultati dello studio di Fase 3 ATTRibute-CM, che hanno dimostrato:
- una riduzione del 42% della mortalità complessiva e degli eventi di ricovero cardiovascolare ricorrente al mese 30
- una riduzione del 50% della frequenza cumulativa degli eventi di ricovero cardiovascolare
- un beneficio rapido con evidenza di differenza rispetto al placebo già dopo 3 mesi
BEYONTTRA è ora approvato negli Stati Uniti, nell'UE, nel Regno Unito e in Giappone. In base a un accordo di collaborazione con Bayer, che si occuperà della commercializzazione nel Regno Unito, BridgeBio riceverà royalty a scaglioni a partire da una percentuale bassa intorno al 30% sulle vendite.
BridgeBio Pharma (BBIO) ha recibido la aprobación en el Reino Unido para BEYONTTRA® (acoramidis), el primer estabilizador de TTR casi completo (≥90%) para el tratamiento de ATTR-CM (amiloidosis por transtiretina con miocardiopatía). La aprobación se basa en los resultados del estudio de Fase 3 ATTRibute-CM, que demostraron:
- una reducción del 42% en la mortalidad total combinada y en las hospitalizaciones recurrentes relacionadas con enfermedades cardiovasculares a los 30 meses
- una reducción del 50% en la frecuencia acumulada de hospitalizaciones cardiovasculares
- un beneficio rápido que mostró diferencia respecto al placebo en tan solo 3 meses
BEYONTTRA está ahora aprobado en EE. UU., UE, Reino Unido y Japón. Bajo un acuerdo de colaboración con Bayer, que gestionará la comercialización en el Reino Unido, BridgeBio recibirá regalías escalonadas que comienzan en un porcentaje bajo cercano al 30% sobre las ventas.
BridgeBio Pharma (BBIO)가 영국에서 BEYONTTRA®(아코라미디스)에 대한 승인을 받았습니다. 이는 ATTR-CM(심근병증을 동반한 트랜스티레틴 아밀로이드증) 치료를 위한 최초의 거의 완전한 TTR 안정제(≥90%)입니다. 이번 승인은 3상 ATTRibute-CM 연구 결과를 기반으로 하며, 다음과 같은 결과를 보여주었습니다:
- 30개월 시점에서 전체 사망률 및 재발성 심혈관계 입원 사건 복합 지표 42% 감소
- 심혈관계 입원 사건 누적 빈도 50% 감소
- 플라시보와의 차이가 빠르게 나타나 3개월 만에 효과 확인
BEYONTTRA는 현재 미국, EU, 영국, 일본에서 승인받았습니다. Bayer와의 협력 계약에 따라 Bayer가 영국 내 상업화를 담당하며, BridgeBio는 매출에 대해 30% 초반대의 단계별 로열티를 받게 됩니다.
BridgeBio Pharma (BBIO) a obtenu l'approbation au Royaume-Uni pour BEYONTTRA® (acoramidis), le premier stabilisateur TTR quasi complet (≥90%) pour le traitement de l'ATTR-CM (amylose à transthyrétine avec cardiomyopathie). Cette approbation repose sur les résultats de l'étude de phase 3 ATTRibute-CM, qui ont démontré :
- une réduction de 42 % de la mortalité toutes causes confondues et des hospitalisations cardiovasculaires récurrentes à 30 mois
- une réduction de 50 % de la fréquence cumulée des hospitalisations cardiovasculaires
- un bénéfice rapide avec une séparation par rapport au placebo dès 3 mois
BEYONTTRA est désormais approuvé aux États-Unis, dans l'UE, au Royaume-Uni et au Japon. Dans le cadre d'un accord de collaboration avec Bayer, qui prendra en charge la commercialisation au Royaume-Uni, BridgeBio percevra des redevances échelonnées débutant dans la tranche basse des trente pour cent sur les ventes.
BridgeBio Pharma (BBIO) hat die Zulassung im Vereinigten Königreich für BEYONTTRA® (Acoramidis) erhalten, den ersten nahezu vollständigen TTR-Stabilisator (≥90%) zur Behandlung von ATTR-CM (Transthyretin-Amyloidose mit Kardiomyopathie). Die Zulassung basiert auf den Ergebnissen der Phase-3-Studie ATTRibute-CM, die Folgendes zeigten:
- 42% Reduktion der kombinierten Gesamtmortalität und wiederkehrender kardiovaskulärer Krankenhausaufenthalte nach 30 Monaten
- 50% Reduktion der kumulativen Häufigkeit kardiovaskulärer Krankenhausaufenthalte
- Schneller Nutzen mit einer Abgrenzung zum Placebo bereits nach 3 Monaten
BEYONTTRA ist nun in den USA, der EU, dem Vereinigten Königreich und Japan zugelassen. Im Rahmen einer Kooperationsvereinbarung mit Bayer, die die Vermarktung im Vereinigten Königreich übernimmt, erhält BridgeBio gestaffelte Lizenzgebühren, beginnend im niedrigen Dreißiger-Prozentbereich auf den Umsatz.
- First-in-class near-complete TTR stabilizer (≥90%) approved in all major markets
- Superior clinical efficacy with 42% reduction in mortality and hospitalization events
- Rapid onset of benefits within 3 months of treatment
- Significant royalty stream starting in low-thirties percent from UK sales
- Commercialization rights in Europe licensed to Bayer, limiting direct revenue potential
- Faces established competition in the ATTR-CM market
Insights
UK approval of BEYONTTRA expands BridgeBio's global reach with strong royalty structure while Bayer handles commercialization costs.
BridgeBio has secured UK regulatory approval for BEYONTTRA® (acoramidis), completing a sweep of authorizations across all major pharmaceutical markets including the US, EU, and Japan. This approval represents a critical regulatory milestone in BridgeBio's global commercialization strategy for their ATTR-CM treatment.
The approval triggers BridgeBio's partnership with Bayer, established in March 2024, which grants Bayer exclusive commercialization rights while BridgeBio will receive royalties beginning in the low-thirties percent on UK sales. This arrangement creates a favorable economic structure for BridgeBio - capturing significant value without bearing the commercial infrastructure costs in the European market.
BEYONTTRA's differentiated positioning is noteworthy. It's labeled across all markets as a near-complete TTR stabilizer (≥90%), establishing a unique efficacy profile. The Phase 3 ATTRibute-CM data demonstrated rapid clinical benefits (within 3 months) with 42% reduction in composite mortality and hospitalization events and 50% reduction in hospitalization frequency at 30 months.
For ATTR-CM, a progressive fatal heart condition with treatment options, this approval addresses significant unmet medical needs while expanding BridgeBio's commercial footprint. The consistent labeling across global markets (highlighting ≥90% TTR stabilization) creates uniform messaging that may streamline marketing efforts and medical education.
BEYONTTRA demonstrates rapid clinical benefits and near-complete TTR stabilization for ATTR-CM patients, filling critical treatment gap in UK healthcare.
BEYONTTRA (acoramidis) represents a significant therapeutic advancement for UK patients with ATTR-CM, a fatal cardiac condition characterized by amyloid deposition in heart tissue. The drug's near-complete transthyretin stabilization (≥90%) addresses the underlying disease mechanism more effectively than previous options.
The clinical profile is particularly compelling. The Phase 3 ATTRibute-CM study showed the most rapid benefit seen in any ATTR-CM trial to date, with treatment effect emerging within just 3 months. This is crucial for a progressive disease where early intervention impacts trajectory. The 42% reduction in all-cause mortality and recurrent cardiovascular hospitalizations and 50% reduction in hospitalization events at 30 months demonstrate meaningful impact on patient outcomes.
ATTR-CM remains underdiagnosed in clinical practice due to nonspecific symptoms and awareness. Dr. Julian Gillmore from University College London's Centre for Amyloidosis notes this often leads to delayed diagnosis and treatment, worsening prognosis. Having another effective disease-modifying treatment option addresses this critical gap in cardiac care.
The medication's mechanism aligns with recent scientific findings linking TTR stability to reduced mortality risk in the general population, providing additional validation for the therapeutic approach. For UK cardiologists managing this challenging condition, BEYONTTRA offers a valuable addition to the treatment armamentarium.
- The UK approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date
- In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placebo
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- Acoramidis is the first and only approved ATTR-CM treatment in the U.S., EU, UK and Japan that all have a label specifying near-complete stabilization (≥
- Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1
- BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of Beyonttra in the UK
PALO ALTO, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the Medicines and Healthcare products Regulatory Agency has granted marketing authorization in the United Kingdom (UK) for acoramidis, under the brand name BEYONTTRA®, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered near-complete (≥
“ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients’ quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied and associated with low awareness, frequently resulting in delayed or completely missed diagnosis,2 which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalizations and escalating healthcare costs and is ultimately fatal,3-5” said Julian Gillmore, M.D., Ph.D., University College London's Centre for Amyloidosis, UK. “The UK authorization of Beyonttra is welcome news for eligible patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.”
The approval in the UK is based on results of the pivotal ATTRibute-CM Phase 3 study of acoramidis, which showed clear benefits on cardiovascular outcomes. ATTRibute-CM evaluated the efficacy and safety of acoramidis in 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR who were randomized 2:1 to receive acoramidis or placebo for 30 months. The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients.
“We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to BEYONTTRA since they are in great need of new disease-modifying treatments for their condition,” said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. “We appreciate the time and commitment of every clinical trial participant and their families, and the dedicated support of the physicians and scientists involved in the clinical program. This important milestone would not have been possible without their commitment to the program. We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world."
Acoramidis was approved as Attruby™ by the U.S. FDA in November 2024 and was approved as BEYONTTRA by the European Commission in February 2025 and the Japanese Ministry of Health, Labour, and Welfare (MHLW) Agency in March 2025 with all labels specifying near-complete stabilization of TTR.
In March 2024, BridgeBio and Bayer initiated a collaboration for acoramidis, which granted Bayer exclusive commercialization rights in Europe. Based on terms of the licensing agreement, BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in the UK following initiation of commercialization efforts.
1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102.
2Rintell et al. Orphanet J Rare Dis. (2021) 16:70. https://doi.org/10.1186/s13023-021-01706-7
3Rozenbaum MH, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiology and therapy. 2021;10:141-59.
4Mallus MT and Rizzello V. Treatment of amyloidosis: present and future. 2023;21;25(Suppl B):B99-B103.
5Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated 27 April 2023. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK574531/ Last accessed: March 2025.
About BEYONTTRA
BEYONTTRA is an orally administered near-complete (≥
About Attruby™ (acoramidis)
INDICATION
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
Diarrhea (
▼: This medicine is subject to additional monitoring. This will allow quick identification of new safety information.
About BridgeBio
BridgeBio is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio Forward-Looking Statements
This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the impact of Beyonttra on clinical outcomes; and the potential benefits of Beyonttra, including its ability to reduce cardiovascular-related hospitalization, improve survival, and preserve functional capacity and quality of life, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies, as reflected in or suggested by these forward-looking statements, are reasonable, BridgeBio can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties, and assumptions, including, but not limited to, the risks associated with BridgeBio’s dependence on third parties for development, manufacture, and commercialization activities related to Beyonttra; government and third-party payor actions; risks and uncertainties relating to competitive products and other changes that may limit demand for Beyonttra; the risk that regulatory authorities may require additional studies or data to support the continued commercialization of Beyonttra; the risk that drug-related adverse events may be observed during commercialization or clinical development; the risk that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review, or approval; the risk of other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of BridgeBio’s filings (such as clinical trial designs, conduct, and methodologies), or the sufficiency of data submitted; the continuing success of its collaborations, including compliance with applicable regulations for the purchase, distribution, storage, export, and sale of active pharmaceutical ingredients and medicinal products; uncertainty regarding any impacts due to global health emergencies, including delays in regulatory review, manufacturing, and supply chain interruptions; adverse effects on healthcare systems and disruption of the global economy; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip; and increasing rates of inflation and changing interest rates on BridgeBio’s business operations and expectations. These risks, as well as those set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission, should be carefully considered. Moreover, BridgeBio operates in a highly competitive and rapidly changing environment, in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise.
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FAQ
What are the key clinical benefits of BEYONTTRA (BBIO) shown in the Phase 3 ATTRibute-CM trial?
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