AKCEA-TTR-LRx

The European Commission has approved WAINZUA (eplontersen), developed by Ionis Pharmaceuticals and AstraZeneca, for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or 2 disease.

Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis.

The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), a heart condition leading to heart failure and death.

BridgeBio's acoramidis, a TTR stabilizer, awaits FDA approval and could compete with Pfizer's tafamidis in the $5.2 billion ATTR-CM market.

Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and Europe for ATTRv-PN treatment.