Phase 2 open-label extension study of patisiran, an investigational siRNA agent for hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN)

Hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP), is an inherited, progressive disease that can cause sensory, motor, and autonomic dysfunction, resulting in significant disability and death. Patisiran is an investigational, systemically administered small interfering RNA (siRNA) targeting wild-type and mutant TTR. A recently completed multi-center, multi-dose Phase 2 trial of patisiran in hATTR-PN patients (N  = 29) showed>80% sustained mean knockdown of serum TTR when administered at a dose of 0.3  mg/kg every 3 weeks with a generally favorable safety profile.

Source: Neuromuscular Disorders - September 10, 2016 Category: Neurology Authors: I. Conceicao, O. Suhr, T. Coelho, M. Waddington Cruz, H. Schmidt, J. Buades, J. Campistol, J. Pouget, J. Berk, D. Adams Source Type: research