Acoramidis approved for heart condition treatment

MHRA authorises drug for adults with rare heart muscle damage

The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light to acoramidis (Beyonttra) for adult patients suffering from cardiomyopathy caused by variant or wild-type transthyretin amyloidosis (ATTR-CM).

The approval follows a fast-track process after the European Medicines Agency (EMA) gave its nod earlier this year.

ATTR-CM occurs when a protein called transthyretin (TTR) malfunctions, leading to the formation of amyloid deposits in the heart. These deposits stiffen the heart muscle, hindering its normal function. Acoramidis hydrochloride, the active ingredient, works by stabilising the TTR protein, thus slowing down the disease’s progression.

Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said: “Keeping patients safe and enabling their access to high quality safe and effective medical products are key priorities for us.”

He added: “The approval of acoramidis reflects our ongoing commitment to ensuring quicker access to medicines that can make a real difference to people suffering from serious diseases and that have proven safety quality and efficacy recognised by comparable international regulators.”

Around 600 people in the UK are estimated to have wild-type ATTR-CM, predominantly older men, while approximately 200 have the hereditary form. The approval was backed by a phase 3 international study involving 632 patients. Results showed acoramidis was significantly more effective than a placebo in slowing heart damage.

Participants taking acoramidis had a 77% higher chance of experiencing a benefit regarding mortality and cardiovascular hospitalisations over 30 months.

The study also tracked patients’ quality of life, TTR levels and other heart markers. Common side effects included diarrhoea and gout. The MHRA will continue to monitor the drug’s safety.