Alnylam's RNAi drug vutrisiran cuts mortality in ATTR-CM by 33% - FDA submission planned

By Clara Rodriguez Fernandez

- Last updated on GMT

© Getty Images
© Getty Images

Related tags cell and gene therapies Phase 3 Clinical trial Pharmacology

Alnylam’s experimental drug vutrisiran has shown promise for the treatment of transthyretin amyloidosis (ATTR) in a phase 3 clinical trial.

The results of the phase 3 trial revealed that vutrisiran reduced the mortality and recurrent cardiovascular events by up to 33% in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). Based on these positive results, Alnylam plans to file a New Drug Application (NDA) with the FDA later this year.

The HELIOS-B clinical study enrolled 654 patients across 79 sites around the globe over a period of up to 36 months. All ATTR patients had a history of heart failure with at least one prior hospitalization.

“The results showed that vutrisiran improved cardiovascular outcomes, including survival, function and quality of life in all patient groups with ATTR cardiomyopathy. We are moving with urgency to file these compelling data with regulators to bring this medicine to patients around the world,” said Pushkal Garg, chief medical Officer of Alnylam.

ATTR - a debilitating and fatal disease

ATTR is a debilitating and fatal disease driven by the accumulation of amyloid deposits across the body, which can affect the heart, nerves and gastrointestinal tract. The disease is caused by mutations in the gene encoding for transthyretin proteins, which lead the proteins to misfold and form amyloid deposits.

RNAi therapeutics such as vutrisiran are designed to block the production of disease-causing proteins by targeting their mRNA precursors. In this case, vutrisiran can inhibit the production of misfolded transthyretin proteins in the liver to prevent the formation of amyloid deposits. The drug is delivered to patients subcutaneously every three months.

Alnylam is a pioneer in the development of RNAi therapeutics. The company’s drug patisiran was the first RNAi treatment to ever receive FDA approval back in 2018. Since then, Alnylam has received approval for multiple RNAi therapies that address diseases with unmet medical needs. 

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