Skip to main content
Premium Trial:

Request an Annual Quote

To Interfere

Stat News heralds the arrival of the "age of RNA interference drugs," as Alnylam Pharmaceuticals has reported positive results from its phase 3 clinical trial of an RNAi-based drug to treat a nerve disorder.

According to Xconomy, Alnylam's 225-patient trial found that the drug, called patisiran, slowed the decline of neurological function that occurs in familial amyloid polyneuropathy, as compared to placebo. FAP is caused by the misfolding and accumulation of the TTR protein, and patisiran targets the gene encoding the protein to turn it off, Stat News says.

Alnylam has also touted the safety profile of patisiran, Xconomy adds. Stat News notes that the clean safety profile has assuaged nerves worried about toxicity, which it says has been reported with other drugs in the company's pipeline.

Stat News also says Alnylam and Sanofi, with which it has partnered, plan to seek approval for the drug in the US by the end of the year and in Europe next year. If approved, patisiran would be the first RNA interference-based commercial product, it notes. Xconomy adds, though, that the drug is likely to be pricy.