UK MHRA authorises BridgeBio's acoramidis for ATTR-CM

Yahoo

29-04-2025

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for BridgeBio Pharma's acoramidis under the name Beyonttra, for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Acoramidis is an orally administered selective small molecule that stabilises transthyretin (TTR) by more than 90%.
This authorisation was supported by the outcomes from the pivotal ATTRibute-CM Phase III trial, which demonstrated cardiovascular benefits.
In the trial, the therapy's safety and efficacy were assessed in 632 symptomatic ATTR-CM subjects, and they were given either the therapy or a placebo for a 30-month period.
The primary clinical endpoints were met at month 30, showing a decrease in cardiovascular-related hospitalisations, preservation of functional capacity, improved survival and quality of life for subjects.
The therapy received the US Food and Drug Administration's (FDA) approval under the name Attruby in November 2024, and as Beyonttra by the European Commission (EC) in February 2025.
It was also approved in Japan by the Ministry of Health, Labour, and Welfare in March 2025.
In the UK, Bayer will manage all commercial activities for the therapy.
In March 2024, the company and Bayer entered a partnership granting the latter exclusive rights to commercialise the therapy in Europe.
According to the licencing agreement, BridgeBio will obtain tiered royalties starting in the low-thirties percent on UK sales after commercialisation efforts commence.
ATTR-CM is a fatal condition that causes heart failure due to restrictive cardiomyopathy.
BridgeBio Cardiorenal chief medical officer and president Jonathan Fox stated: 'We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to Beyonttra since they are in great need of new disease-modifying treatments for their condition.
'We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world."
"UK MHRA authorises BridgeBio's acoramidis for ATTR-CM" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand.
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