Acoramidis: FDA Authorizes First Near-Total Stabilizer for ATTR Cardiomyopathy

 Acoramidis has been approved by the US FDA for treating ATTR cardiomyopathy (ATTR-CM) in adults.

This approval is based on the ATTRibute-CM trial, which demonstrated cardiovascular benefits with acoramidis.

BridgeBio has pledged to provide acoramidis free for life to patients who participated in the trial.

The pricing for other patients is set at $244,000 annually, sparking concerns over affordability.

The only other approved drug for ATTR-CM, tafamidis, was priced at $268,000 per year when launched in 2019.

A report from the ICER suggests transthyretin-stabilizing agents need an 85-95% price reduction to meet cost-effectiveness standards.

In the ATTRibute-CM trial, patients taking acoramidis saw significant improvement in survival, cardiovascular hospitalization, and functional capacity at 30 months.

Acoramidis mimics a naturally occurring mutation of the TTR gene, targeting the root cause of ATTR-CM.

The drug achieves “near-complete TTR stabilization,” addressing disease progression effectively.

BridgeBio plans to seek approval for acoramidis in Europe, Japan, and Brazil.

A new therapy, nexiguran ziclumeran, using CRISPR-Cas9, showed promise in phase I trials for ATTR-CM.

Take-Home Points:

1. Acoramidis is the second FDA-approved drug for ATTR-CM, offering significant clinical benefits.
2. Its price point of $244,000 per year raises concerns over affordability and cost-effectiveness.
3. The drug targets the root cause of ATTR-CM by achieving “near-complete TTR stabilization.”
4. BridgeBio is expanding its approval strategy to global markets, including Europe, Japan, and Brazil.
5. Gene-editing therapy, nexiguran ziclumeran, offers a potential future treatment option for ATTR-CM.