Acoramidis Receives Positive CHMP Opinion for Treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
- 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
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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
- 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 (≥90%) transthyretin (TTR) stabilizer. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. In the Phase 3 study ATTRibute-CM, acoramidis showed clear benefits on cardiovascular outcomes.
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 (≥90%) stabilizer of
Transthyretin (TTR) approved in the U.S. for the treatment of
adult patients with ATTR-CM to reduce cardiovascular death and
cardiovascular-related hospitalization. Attruby was generally
well-tolerated. The most common side effects were mild and
included diarrhea and abdominal pain that were resolved without
drug discontinuation. BridgeBio offers an extensive suite of
programs to help patients access our medicines.
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.
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