United States: The U.S. Food and Drug Administration approved a new medication to treat a critical heart disorder that impacts many people across the country, as reported by HealthDay.
Amvuttra developed by Alnylam Pharmaceuticals functions as a treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
The American Heart Association (AHA) defines ATTR-CM as a heart condition where harmful proteins accumulate leading to cardiac dysfunction that hinders blood pumping function.
Clinical Trials Show Promising Results
Laboratory tests show Amvuttra reduced mortality and cardiovascular events by 28% better than a dummy substance over a period of three years. Patients receive this medication through an injection that they should take three months apart.
“We hope this could become our flagship franchise. We’re going to help thousands more patients,” Alnylam CEO Yvonne Green street said in an interview with STAT News. “It’s also a transformational moment for the company.”
Amvuttra approved to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. https://t.co/sFbSYyPXSb
— MPR (@eMPR) March 22, 2025
A High Price Tag Raises Accessibility Concerns
The medical intervention has an extremely high financial consequence. Annual expenses regarding the treatment amount to $476,000, with each dose priced at $119,000. Readers will find this price at least twice higher than what BridgeBio charges for its equivalent drug while being beyond the range of Pfizer’s pill therapy.
“There are many, many, many undiagnosed patients and substantial unmet needs in this space. This market is very large, underdiagnosed, undertreated,” said Dr. Mani Foroohar, an analyst with the healthcare investment company Leerink.
The steep price of the drug creates barriers for patients whose insurance coverage includes the Medicare Advantage benefit plan to access the medication. Private payers typically choose Pharmaceutical Corporation Pfizer’s pills and BridgeBio’s products for cost reasons.
This medication differs in its operation from other drugs. RNA technology utilized by Amvuttra creates a blockade that prevents protein manufacturing while the other treatments attempt to secure agarose proteins from causing damage.
How Amvuttra Compares to Other Treatments
The clinical research conducted by BridgeBio revealed their treatment reduced mortality and heart hospital admissions by 42% throughout 30 months.
Results from Amvuttra’s studies indicated a 28% reduction in patient outcomes during the three-year period. Due to different testing methods, there exists an obstacle when attempting to match the outcomes of these medical trials directly, as reported by HealthDay.
Expanding Access to ATTR-CM Patients
The healthcare community has approved Amvuttra treatment for nerve-associated ATTR patients. The latest approval from regulatory bodies enables Alnylam to reach more patients with their medication.