- BridgeBio Pharma (BBIO, Financial) secures UK approval for BEYONTTRA® (acoramidis) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
- Phase 3 study shows BEYONTTRA offers a 42% reduction in mortality and cardiovascular hospitalizations, with rapid benefits visible within 3 months.
- BridgeBio collaborates with Bayer for UK commercialization, receiving royalties starting in the low-thirties percent on sales.
BridgeBio Pharma, Inc. (BBIO) has received marketing authorization from the UK's Medicines and Healthcare products Regulatory Agency for BEYONTTRA® (acoramidis), a near-complete transthyretin (TTR) stabilizer designed for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults. This approval marks a significant milestone, as BEYONTTRA is now approved in all major pharmaceutical markets, including the US, EU, and Japan.
The approval is grounded in the positive outcomes of the Phase 3 ATTRibute-CM study, which highlighted BEYONTTRA’s efficacy by achieving a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events over a 30-month period. Furthermore, the study demonstrated a 50% reduction in the cumulative frequency of cardiovascular hospitalizations. Notably, the treatment effects of BEYONTTRA were evident within just 3 months, a rapid benefit compared to the placebo group.
Under the terms of a collaboration agreement with Bayer, BridgeBio will benefit from tiered royalties on UK sales, commencing in the low-thirties percent range. Bayer will manage all commercialization activities in the UK, allowing BridgeBio to capitalize on sales while minimizing infrastructure costs.
BEYONTTRA's approval provides a vital treatment option for patients with ATTR-CM, a progressive and fatal condition often resulting in heart failure. The drug's mechanism targets near-complete stabilization (?90%) of TTR, addressing the disease's underlying pathology more effectively than previous treatments. Dr. Julian Gillmore from University College London's Centre for Amyloidosis underscores the significance of this approval in bridging critical treatment gaps in the UK healthcare system.
