BridgeBio Pharma, Inc. (NASDAQ:BBIO) received U.S. Food and Drug Administration (FDA) approval for Attruby™ (acoramidis), an oral medication for the treatment of adults with ATTR-CM aimed at reducing cardiovascular death and hospitalization. The approval followed positive results from the ATTRibute-CM Phase 3 study. In response to the news, BridgeBio shares climbed by 25%.
The ATTRibute-CM study demonstrated that Attruby significantly curtailed death and cardiovascular-related hospitalization and enhanced the quality of life for patients. Attruby stands out as the first and only product approved with a label that specifies near-complete stabilization of Transthyretin (TTR), a protein associated with ATTR-CM.
The Phase 3 trial involved 632 participants and met its primary endpoint with a Win Ratio of 1.8, indicating a statistically significant treatment effect. Attruby's impact was evident in the Kansas City Cardiomyopathy Questionnaire and a 6-minute walk test, showing a notable improvement in patient outcomes.
The approval of Attruby has been met with enthusiasm from medical professionals and patient support groups. Muriel Finkel, President of Amyloidosis Support Groups, expressed hope and opportunity for patients with amyloidosis, emphasizing the importance of new treatments for better outcomes.
Martha Grogan, M.D., from the Mayo Clinic, highlighted the significance of having a new treatment that offers excellent TTR stabilization and improves patient outcomes, potentially transforming a fatal disease into a manageable condition.
BridgeBio also offers ForgingBridges™, a patient support services program in the U.S., designed to assist patients prescribed Attruby and their families in accessing the therapy, including insurance resources and financial assistance.
Looking forward, BridgeBio's CEO, Neil Kumar, Ph.D., expressed gratitude to those involved in the trial and conveyed the company's intention to seek approvals globally, with the next targets being Europe, Japan, and Brazil. BridgeBio has already submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025, and has granted Bayer (OTC:BAYRY) the exclusive rights to commercialize acoramidis in Europe.
In light of these developments, Scotiabank (TSX:BNS) analyst Greg Harrison raised the price target on BridgeBio Pharma to $48.00 from $45.00 while maintaining a Sector Outperform rating.
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