Alnylam Wins FDA Approval for AMVUTTRA® to Treat Cardiomyopathy

Alnylam Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its RNAi-based treatment, AMVUTTRA® (vutrisiran).

ATTR-CM is a severe and rapidly progressing condition that affects approximately 150,000 people in the United States and over 300,000 globally. It leads to the deposition of misfolded transthyretin (TTR) fibrils, causing irreversible organ damage and significantly reducing life expectancy. 

Many patients remain undiagnosed or experience disease progression despite treatment with available therapies.

AMVUTTRA is designed to address the underlying cause of ATTR-CM by reducing the production of TTR through RNAi technology. It is administered via a subcutaneous injection four times a year, aiming to prevent the accumulation of harmful amyloid deposits in the heart.

Transthyretin amyloidosis (ATTR) is a progressive and often fatal disease caused by the accumulation of misfolded TTR proteins, leading to damage in the heart, nerves, and other organs. The condition exists in hereditary (hATTR) and wild-type (wtATTR) forms, affecting tens of thousands of patients worldwide.

RNA interference (RNAi) is a biological process that prevents the production of disease-causing proteins by silencing messenger RNA (mRNA). This mechanism has led to the development of RNAi-based treatments like AMVUTTRA, which target the root cause of diseases rather than just managing symptoms.

The FDA’s approval of AMVUTTRA for ATTR-CM represents an important step in improving treatment options for patients with this serious condition.

The approval extends the drug’s indication to include the treatment of cardiomyopathy associated with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults, aiming to reduce cardiovascular mortality, hospitalisations, and urgent heart failure visits. 

With this decision, AMVUTTRA becomes the first FDA-approved therapy for both ATTR-CM and hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN) in adults.

The approval is supported by data from the HELIOS-B Phase 3 clinical trial, which assessed AMVUTTRA’s impact on ATTR-CM patients. The study showed a statistically significant reduction in the risk of cardiovascular-related deaths and hospitalisations compared to a placebo. 

Alnylam has outlined plans to ensure broad access to AMVUTTRA, with insurance coverage expected to be similar to that of its use in hATTR-PN. 

The company also provides patient support services, including financial assistance and treatment guidance through its Alnylam Assist® programme.

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