Alnylam's patisiran backed by FDA advisory committee for ATTR cardiomyopathy

Alnylam Pharmaceuticals has announced that its RNAi therapeutic, patisiran, has been backed by a US Food and Drug Administration (FDA) panel of experts to treat a type of heart disease connected with a rare organ-damaging disorder.

The FDA's Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted nine to three that the benefits of the drug outweigh its risks for treating patients with cardiomyopathy induced by transthyretin (ATTR) amyloidosis.

ATTR淀粉样变是一个诊断和快速progressive disease caused by misfolded transthyretin proteins, which accumulate as amyloid deposits in various parts of the body, including the heart, resulting in cardiomyopathy and heart failure.

The cardiac manifestations associated with ATTR can be debilitating, and treatment options are limited, with many patients continuing to progress on, or unable to access, the current standard-of-care.

Patisiran is designed to target and silence transthyretin messenger RNA, thereby reducing the production of the protein before it is made.

The CRDAC’s decision was supported by positive results from the late-stage APOLLO-B study, which demonstrated favourable effects on functional capacity and health status, and quality of life in patients relative to placebo.

The FDA will now consider the panel’s advice as it makes a decision regarding the potential approval of Patisiran in this patient population, with an action date set for 8 October.

Patisiran is already approved in multiple countries, including the US, to treat the polyneuropathy of hereditary ATTR amyloidosis in adults.

Alnylam’s chief medical officer, Pushkal Garg, said: “The positive outcome of [the] meeting is supported by the efficacy and safety data observed in the APOLLO-B phase 3 study, and is another step toward bringing patients with the cardiomyopathy of ATTR amyloidosis a novel treatment option that addresses the underlying cause of disease and has the potential to meaningfully benefit patients’ functional capacity and quality of life.”

In July, Alnylampresented positive resultsfor its Regeneron-partnered gene silencing RNAi therapeutic in early-onset Alzheimer’s disease.

Interim results from an early-stage study showed that a single injection of ALN-APP, which is designed to switch off the production of amyloid precursor protein (APP) in the central nervous system, was able to rapidly reduce levels of the protein, with clinical effect sustained over six months.