BridgeBio gets EU nod for ATTR cardiomyopathy drug

The European Commission has approved BridgeBio's acoramidis for life-threatening heart disorder transthyretin amyloidosis cardiomyopathy (ATTR-CM), setting up a market clash with Pfizer.

The oral drug – which has been cleared under the Beyonttra trade name – can be used to treat cardiomyopathy in wild-type or variant ATTR patients and will be launched by European marketing partner Bayer in the second half of this year.

The EU is the second market for the drug after the US, where it was approved under the Attruby brand name last November, on the back of clinical data showing that it was able to reduce cardiovascular death and cardiovascular-related hospitalisation in adults with ATTR-CM, a progressive disease that leads to heart failure and death.

It is expected to be a major competitor to Pfizer's blockbuster Vyndamax/Vyndaqel/Vynmac franchise – based on tafamidis – which has been approved for ATTR-CM since 2019 and is also used to treat polyneuropathy associated with ATTR (ATTR-PN). Last year, sales of the Vyndaqel family grew 65% to $5.45 billion.

Both acoramidis and tafamidis are transthyretin (TTR) stabilisers, designed to block the formation of amyloid fibrils in tissues and organs that are a hallmark of ATTR and responsible for symptoms.

According to BridgeBio, its drug has shown the most rapid benefit in ATTR-CM of any therapy tested in phase 3 trials and is the only one with a label specifying near-complete stabilisation (≥90%) of TTR, although, Pfizer disputes that interpretation claiming its drug has shown similar levels in trials.

Early signs from the US market have been positive, with the Palo Alto, California-based company reporting that 430 prescriptions for Attruby had been written before the end of 2024.

The drug had a US list price at launch of around $244,000 per year based on the recommended dose of two 356mg tablets taken twice daily, which was a discount on the $268,000 annual cost of the Vyndaqel range.

BridgeBio now stands to receive a $75 million milestone payment from Bayer as a result of the EU approval and will book royalties on sales by Bayer at a rate in the low-30s percentage or higher.

The company is also anticipating approval of acoramidis in the first half of this year in Japan, where it is licensed to AstraZeneca.

Another rival in the ATTR-CM market could be on the way, as Alnylam is waiting to hear from the FDA whether its gene-silencing therapy vitrusiran – already approved as Amvuttra for ATTR-PN – will get its US label extended to include ATTR-CM. That decision is due by 23rd March.