Acoramidis Receives Positive CHMP Opinion for Treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
BridgeBio Pharma (BBIO) announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended approval of acoramidis in the EU for treating transthyretin amyloid cardiomyopathy (ATTR-CM). The recommendation follows positive Phase 3 ATTRibute-CM study results, showing a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events, and a 50% reduction in cumulative frequency of cardiovascular-related hospitalization events at Month 30.
The drug was already approved by the FDA on November 22, 2024, as Attruby™, becoming the first ATTR-CM treatment achieving near-complete stabilization in the US. Through a collaboration with Bayer, BridgeBio holds US marketing rights while Bayer manages European distribution. Following expected European Commission approval, Bayer plans to launch acoramidis in Europe in first half 2025.
BridgeBio Pharma (BBIO) ha annunciato che il Comitato per i Medicinali per Uso Umano (CHMP) ha raccomandato l'approvazione dell'acoramidis nell'UE per il trattamento della cardiomiopatia amiloide da transtiretina (ATTR-CM). La raccomandazione segue i risultati positivi dello studio di fase 3 ATTRibute-CM, che mostrano una riduzione del 42% della mortalità complessiva e degli eventi di ospedalizzazione ricorrenti legati a patologie cardiovascolari, e una riduzione del 50% della frequenza cumulativa degli eventi di ospedalizzazione legati a patologie cardiovascolari al mese 30.
Il farmaco è già stato approvato dalla FDA il 22 novembre 2024, come Attruby™, diventando il primo trattamento per l'ATTR-CM a raggiungere una stabilizzazione quasi completa negli Stati Uniti. Attraverso una collaborazione con Bayer, BridgeBio detiene i diritti di marketing negli Stati Uniti mentre Bayer gestisce la distribuzione in Europa. Dopo l'attesa approvazione della Commissione Europea, Bayer prevede di lanciare l'acoramidis in Europa nella prima metà del 2025.
BridgeBio Pharma (BBIO) anunció que el Comité de Medicamentos para Uso Humano (CHMP) ha recomendado la aprobación de acoramidis en la UE para el tratamiento de la cardiomiopatía amiloide por transtiretina (ATTR-CM). La recomendación sigue los resultados positivos del estudio de fase 3 ATTRibute-CM, que muestran una reducción del 42% en la mortalidad por todas las causas y en eventos de hospitalización recurrentes relacionados con la cardiovascular, y una reducción del 50% en la frecuencia acumulada de eventos de hospitalización relacionados con la cardiovascular al mes 30.
El fármaco ya fue aprobado por la FDA el 22 de noviembre de 2024, como Attruby™, convirtiéndose en el primer tratamiento para la ATTR-CM que logra una estabilización casi completa en los Estados Unidos. A través de una colaboración con Bayer, BridgeBio tiene los derechos de comercialización en Estados Unidos, mientras que Bayer gestiona la distribución en Europa. Tras la esperada aprobación de la Comisión Europea, Bayer planea lanzar acoramidis en Europa en la primera mitad de 2025.
브리지바이오 제약 (BBIO)는 인간 사용을 위한 의약품 위원회 (CHMP)가 트랜스티레틴 아밀로이드 심근병증 (ATTR-CM) 치료를 위한 아코라미디스의 유럽 연합 내 승인을 추천했다고 발표했습니다. 이 추천은 Phase 3 ATTRibute-CM 연구 결과에 따른 것으로, 42%의 모든 원인에 의한 사망률 및 재발성 심혈관 관련 입원 사건의 감소를 보여주며, 30개월 기준으로 50%의 심혈관 관련 입원 사건의 누적 빈도 감소를 나타냅니다.
이 약물은 이미 2024년 11월 22일 FDA의 승인을 받았으며, Attruby™라는 이름으로 미국에서 거의 완전한 안정성을 달성한 첫 번째 ATTR-CM 치료제가 되었습니다. Bayer와의 협력을 통해 BridgeBio는 미국 내 마케팅 권리를 보유하고 있으며, Bayer는 유럽 내 유통을 관리합니다. 유럽연합 집행위원회의 승인이 예상됨에 따라 Bayer는 2025년 상반기 내에 유럽에서 아코라미디스를 출시할 계획입니다.
BridgeBio Pharma (BBIO) a annoncé que le Comité des médicaments à usage humain (CHMP) a recommandé l'approbation de l'acoramidis dans l'UE pour le traitement de la cardiomyopathie amyloïde à transthyrétine (ATTR-CM). Cette recommandation fait suite aux résultats positifs de l'étude de phase 3 ATTRibute-CM, montrant une réduction de 42% de la mortalité toutes causes confondues et des événements d'hospitalisation cardiovasculaire récurrents, ainsi qu'une réduction de 50% de la fréquence cumulative des événements d'hospitalisation liés à des problèmes cardiovasculaires au mois 30.
Le médicament a déjà été approuvé par la FDA le 22 novembre 2024, sous le nom d'Attruby™, devenant le premier traitement pour l'ATTR-CM à atteindre une stabilisation presque complète aux États-Unis. Grâce à une collaboration avec Bayer, BridgeBio détient les droits de commercialisation aux États-Unis, tandis que Bayer gère la distribution en Europe. Suite à l'approbation attendue de la Commission européenne, Bayer prévoit de lancer l'acoramidis en Europe dans la première moitié de 2025.
BridgeBio Pharma (BBIO) gab bekannt, dass das Ausschuss für Humanarzneimittel (CHMP) die Genehmigung von Acoramidis in der EU zur Behandlung der Transthyretin-Amyloid-Kardiomyopathie (ATTR-CM) empfohlen hat. Die Empfehlung folgt auf die positiven Ergebnisse der Phase 3 ATTRibute-CM-Studie, die eine 42%ige Reduktion der Gesamtmortalität und wiederholter herz-kreislaufbezogener Krankenhausaufenthalte zeigen und eine 50%ige Reduktion der kumulierten Häufigkeit herz-kreislaufbezogener Krankenhausaufenthalte nach 30 Monaten zeigen.
Das Medikament wurde bereits am 22. November 2024 von der FDA als Attruby™ genehmigt und ist somit die erste ATTR-CM-Behandlung, die eine nahezu vollständige Stabilisierung in den USA erreicht hat. Durch eine Zusammenarbeit mit Bayer hält BridgeBio die Vermarktungsrechte in den USA, während Bayer die europäische Verteilung verwaltet. Nach der erwarteten Genehmigung der Europäischen Kommission plant Bayer, Acoramidis in der ersten Hälfte von 2025 in Europa auf den Markt zu bringen.
- FDA approval received for Attruby™ on November 22, 2024
- Positive CHMP recommendation for EU approval
- Phase 3 trial showed 42% reduction in mortality and hospitalization events
- 50% reduction in cardiovascular-related hospitalization frequency
- Strategic partnership with Bayer for European market distribution
- None.
Insights
- The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of acoramidis in the EU based on positive results from the Phase 3 ATTRibute-CM study; final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission within the coming months
- Acoramidis, a near-complete (≥
90% ) stabilizer of transthyretin (TTR), was approved on November 22, 2024, by the U.S. Food and Drug Administration (FDA) as Attruby™ to reduce cardiovascular death and cardiovascular-related hospitalization in ATTR-CM; Attruby™ is the first and only approved ATTR-CM treatment in the United States that achieves near-complete stabilization - In ATTRibute-CM, 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 cardiovasacular-related hospitalization (CVH)) durably separated relative to placebo
- A
42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30 - A
50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30
- In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovasacular-related hospitalization (CVH)) durably separated relative to placebo
- 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
PALO ALTO, Calif., Dec. 13, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization in the European Union (EU) for acoramidis 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 (≥
The final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the coming months. Acoramidis was approved by the FDA on November 22, 2024 as Attruby™, the first and only approved product for adults with ATTR-CM in the United States with a label specifying near-complete stabilization of TTR.
The positive CHMP opinion for acoramidis is based on the positive ATTRibute-CM Phase 3 study results. The study investigated the efficacy and safety of acoramidis given twice daily compared with placebo, in subjects with ATTR-CM. The study met its primary clinical endpoints by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients in need.
“We are encouraged by the CHMP’s positive recommendation of acoramidis and the step forward this represents for the patient community,” said Julie Miller Everett, Chief Business Officer for BridgeBio Cardiovascular. “We are excited to work with Bayer to make acoramidis available to patients in Europe upon approval by the European Commission, which we are looking forward to in early 2025.”
Since March 2024, BridgeBio and Bayer have pursued a collaboration for acoramidis. BridgeBio holds the marketing rights for acoramidis in the U.S., while Bayer holds the marketing rights for the product in Europe. This partnership leverages Bayer’s long legacy of expertise in cardiovascular disease and its established European cardiovascular infrastructure paired with BridgeBio’s leadership in the emerging field of ATTR-CM. Pending European Commission approval, Bayer plans to launch acoramidis in Europe in the first half of 2025.
1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102.
About Attruby™ (acoramidis)
Attruby is the only near-complete (≥
About BridgeBio
BridgeBio is a new type of 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 acoramidis on clinical outcomes, potential benefits of acoramidis, and the potential outcomes and expected timing of regulatory reviews and approvals by the European Medicines Agency and European Commission, 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 those 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, the risks regulatory authorities may require additional studies or data to support commercialization of acoramidis, data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval, 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, and 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, increasing rates of inflation and changing interest rates, on BridgeBio’s business operations and expectations, as well as those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K and Quarterly Report on From 10-Q and its other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very 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 update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
BridgeBio Media Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220
FAQ
What were the key results of BBIO's Phase 3 ATTRibute-CM trial for acoramidis?
When did BBIO receive FDA approval for Attruby (acoramidis)?
What is the current status of BBIO's acoramidis approval in Europe?
When will BBIO's partner Bayer launch acoramidis in Europe?
What is the partnership structure between BBIO and Bayer for acoramidis?
