Revusiran, an investigational ribonucleic acid interference (RNAi) therapeutic, was being developed for the treatment of hereditary ATTR amyloidosis, a rare disease caused by the build-up of a destructive substance called amyloid within the body’s tissues.
Alnylam took the decision after a phase 2 open-label extension study revealed some disconcerting results, with patients who were taking resuviran tending to ie sooner than those on the placebo.
“Patient safety comes first,” said John Maraganore, PhD, chief executive officer at Alnylam.
“We have stopped all dosing and are actively monitoring patients across revusiran studies to ensure their safety. We will also continue to evaluate ENDEAVOUR [phase study] data to understand the potential cause of these findings,” Maraganore said.
“While this outcome is disappointing given the lack of available treatment options for patients suffering from this devastating disease, we remain committed to serving the needs of the ATTR amyloidosis community,” Maraganore said.
Story by ProactiveInvestors