BridgeBio Pharma recently achieved a significant milestone with the FDA's approval of its new drug, Attruby (acoramidis), aimed at treating transthyretin amyloid cardiomyopathy (ATTR-CM). This rare heart condition affects up to 500,000 individuals globally and is characterized by the buildup of misfolded transthyretin proteins, which can lead to severe heart complications, including failure to pump blood effectively.
The FDA approval allows Attruby to be marketed for adult patients grappling with ATTR-CM, promising to reduce both the risk of cardiovascular death and the necessity for hospitalizations due to the disease. Notably, Attruby is the first medication with the FDA label specifying near-complete stabilization of transthyretin (TTR), distinguishing it from existing treatments like Pfizer's Vyndaqel, which also targets this condition.
BridgeBio's approval followed positive results from the ATTRibute-CM clinical trial, which included 632 participants. This study revealed significant benefits for patients taking Attruby over 30 months, as it improved survival rates and reduced hospital visits when compared to patients receiving placebo. The trial's outcomes were published earlier this year, highlighting the drug's potential as not just another treatment but possibly a new standard of care.
CEO Neil Kumar emphasized the importance of this drug, stating, "This is the only oral stabilizer with the verbiage ‘near-complete stabilization’ in the label," which indicates its unique effectiveness at the molecular level. The dual approach of reducing heart failure risk and enhancing the quality of life for patients sets Attruby apart.
Despite its promising launch, BridgeBio will face stiff competition against established drugs like Pfizer's tafamidis, which reported sales exceeding $3.9 billion over the first nine months of 2024. Pfizer's drug has reached widespread acceptance among healthcare providers, making it the go-to treatment option for many patients currently. According to various analysts, BridgeBio's challenge will be not only to attract prescribers to Attruby but to establish its role as either a standalone or complementary treatment for patients who are currently stabilized on Pfizer's offerings.
Along with the competitive marketplace, BridgeBio has set Attruby's wholesale price at approximately $18,759.12 per month—around $244,500 annually. This pricing strategy is slightly lower than Pfizer's tafamidis, but both products remain significantly above the cost-effectiveness range established by the Institute for Clinical and Economic Review, indicating potential financial barriers for patients seeking treatment.
Kumar noted their commitment to ensuring access for patients who participated in the clinical trials, pledging to provide them with the drug free of charge for life. They also recognize the educational component necessary for healthcare providers, as many patients with ATTR-CM remain undiagnosed or misdiagnosed, leading to delayed treatment.
Meanwhile, Alnylam Pharmaceuticals is also preparing to enter the ATTR-CM market. They recently received FDA acceptance for its supplemental new drug application for their already marketed treatment, Amvuttra, targeting the same amyloidosis condition. This growing competition could reshape the market dynamics, emphasizing the need for continued innovation and awareness of ATTR-CM within the medical community.
The approval of Attruby is seen not just as another entry to the pharmaceutical field but as part of the broader developments aimed at tackling this challenging health condition. Kristen Hsu, Executive Director of the Amyloidosis Research Consortium, expressed optimism about this treatment option, stating, “This new therapy provides more choices for patients and healthcare providers.”
Reception among professionals appears cautiously optimistic, with many noting the meaningful advancements it offers compared to existing treatments. Yet, there remains skepticism about whether Attruby can unseat the entrenched presence of Pfizer’s products without the additional label data supporting all-cause mortality benefits.
BridgeBio’s approach to the market will focus on collaboration with healthcare providers to identify patients who have not yet received the diagnosis of ATTR-CM. Kumar mentioned the notable gap between the estimated 250,000 to 300,000 individuals living with the disease and the significantly lower numbers who are currently being treated.
BridgeBio’s aspirations extend beyond the U.S. market, with ambitions for global reach. They are actively pursuing necessary regulatory pathways for approvals expected by 2025 across various regions, including Europe, Japan, and Brazil, where market opportunities for similar treatments are rising.
Overall, the approval of Attruby marks not just the rise of BridgeBio as a noteworthy competitor but signals hope for patients battling cardiomyopathy related to transthyretin amyloidosis. This approval exemplifies the potential for innovation and improved health outcomes for those affected by this challenging condition.
