FDA Approves Alnylam’s First-of-its-Kind Drug AMVUTTRA® to Prevent Heart-Related Deaths
Alnylam Pharmaceuticals has received approval from the U.S. Food and Drug Administration for a supplemental New Drug Application for AMVUTTRA® (vutrisiran). The decision allows AMVUTTRA to be used in the treatment of cardiomyopathy linked to wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. It is now authorised to reduce cardiovascular-related deaths, hospital admissions, and emergency visits due to heart failure.
With this expanded approval, AMVUTTRA becomes the first and only medicine approved in the United States to treat both ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
ATTR-CM is a serious and often fatal condition. It progresses quickly and is caused by the build-up of misfolded transthyretin (TTR) proteins that form damaging fibrils. These deposits can lead to irreversible heart damage and early death. An estimated 150,000 people in the U.S. and over 300,000 globally are affected by the disease. Many individuals remain undiagnosed or untreated, and current treatment options often fail to stop the progression of the disease.
AMVUTTRA is an RNAi therapeutic designed to reduce the production of TTR in the liver, targeting the disease at its source. Administered just four times a year by subcutaneous injection, it leads to a rapid and significant drop in TTR levels. This action slows down or prevents the build-up of harmful fibrils that damage the heart.
Patients on AMVUTTRA also saw better maintenance of physical ability and quality of life. Additionally, improvements were recorded in key heart biomarkers, such as NT-proBNP and troponin I, which are linked to cardiovascular outcomes.
The safety profile of AMVUTTRA remains well-understood, building on data from the earlier HELIOS-A trial for hATTR-PN. Across both trials and more than 5,000 patient-years of exposure, the most frequently reported side effects included pain in the limbs (15%), joint pain (11%), breathlessness (7%), and reduced vitamin A levels (7%).
The approval of AMVUTTRA for ATTR-CM is considered a major step forward in the management of this difficult condition, offering a new and effective treatment approach for affected patients.
