Alnylam Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has decided to proceed with talks to review the supplemental New Drug Application for patisiran (Onpattro®), a promising investigational therapeutic that may be useful in treating cardiomyopathy in hereditary transthyretin amyloidosis (hATTR). 

Patisiran has already gained FDA approval for the treatment of polyneuropathy in adult patients with hATTR. To facilitate discussions about the merits of this therapeutic in treating hATTR-associated cardiomyopathy, a date of September 13, 2023, has been set for the Cardiovascular and Renal Drugs Advisory Committee to meet. 

hATTR is a rare disease in which misfolded TTR proteins accumulate throughout the body. Pathology encompassing the cardiovascular and nervous systems are common. 

Because patisiran has been approval to treat hATTR-associated polyneuropathy, researchers have a reasonable grasp of its safety profile. Approximately 2 in 10 patients on patisiran experience infusion-related reactions, the most common being flushing, back pain, nausea, and headache. To minimize these effects, clinicians recommend that patients receive a corticosteroid, acetaminophen, and antihistamines at least 1 hour prior to drug infusion. 

Read more about hATTR etiology 

Patisiran is a RNA interference therapeutic; this class of drugs enables a natural cellular process of gene silencing. It is an innovative, state-of-the-art method for treating diseases with a genetic component. Patisiran was developed to silence messenger RNAs that encode for disease-causing proteins, which can have a major impact on achieving the goals of disease control and elevating health-related quality of life. 


Alnylam announces date of planned FDA advisory committee meeting for ONPATTRO® (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis. News release. Alnylam Pharmaceuticals; June 30, 2023.