FDA Approves Acoramidis (ATTRuby) for Transthyretin Amyloid Cardiomyopathy

• The FDA has approved acoramidis (ATTRuby) by BridgeBio Pharma for treating transthyretin amyloid cardiomyopathy (ATTR-CM), offering a new treatment option.
• Acoramidis is a near-complete TTR stabilizer (≥90%) and is approved to reduce cardiovascular death and cardiovascular-related hospitalization in ATTR-CM patients.
• BridgeBio is pursuing further approvals in Europe, Japan, and Brazil, with Bayer holding exclusive commercialization rights.
• The approval introduces competition to Pfizer's tafamidis, which has been the primary treatment for ATTR-CM since 2019; Alnylam is also seeking approval for vutrisiran for ATTR-CM.

The U.S. Food and Drug Administration (FDA) has approved acoramidis (ATTRuby), a novel drug developed by BridgeBio Pharma, for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). This approval marks a significant advancement in the treatment landscape for ATTR-CM, providing a new therapeutic option for patients suffering from this life-threatening condition.

Clinical Significance

Acoramidis is designed as a near-complete TTR stabilizer (≥90%) and has demonstrated efficacy in reducing cardiovascular death and cardiovascular-related hospitalizations in patients with ATTR-CM. The approval is based on clinical trial data demonstrating significant improvements in patient outcomes compared to existing treatments or placebo.

Neil Kumar, PhD, founder and CEO of BridgeBio, expressed his gratitude to the trial participants, their families, and the team involved in the drug's development. "Our journey is not over as we look to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment," he stated.

Addressing Unmet Needs in ATTR-CM

ATTR-CM is a progressive disease characterized by the accumulation of misfolded transthyretin (TTR) protein in the heart, leading to heart failure and other cardiovascular complications. Until recently, treatment options have been limited, with Pfizer’s tafamidis being the primary therapy since 2019. The approval of acoramidis introduces much-needed competition and an alternative mechanism of action to the market.

Muriel Finkel, president of Amyloidosis Support Groups, emphasized the importance of new treatment options. "The need for more treatment options for patients living with ATTR-CM is crucial to achieving the goal of better outcomes and improved quality of life," she noted. "Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis."

Competitive Landscape

The ATTR-CM market is becoming increasingly competitive. In addition to BridgeBio's acoramidis, Alnylam Pharmaceuticals has submitted a request to the FDA to approve vutrisiran, already approved for polyneuropathy of hereditary ATTR amyloidosis, as a treatment for ATTR-CM. Pushkal Garg, MD, Chief Medical Officer at Alnylam, stated, "We look forward to working with the FDA over the coming months on this application to bring this medicine to patients as rapidly as possible. Additional filings in other geographies are underway."

Future Directions

BridgeBio is actively pursuing regulatory approval for acoramidis in Europe, Japan, and Brazil. The company has granted exclusive commercialization rights for the drug to Bayer, signaling a strategic partnership to expand the drug's global reach.