Alnylam Pharmaceuticals has started the Phase III open-label extension study (Apollo-Ole) with patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) to treat TTR-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropthy (FAP).
In the current study, the company will enrol all patients who complete the Apollo Phase III trial with patisiran.
Alnylam Pharmaceuticals chief medical officer and R&D executive vice-president Akshay Vaishnaw said: "The initiation of Apollo-Ole marks our continued progress with patisiran, and we are pleased to provide patients previously treated in Apollo with the opportunity to receive patisiran on an ongoing basis.
"The Apollo trial continues enrolling patients with FAP, and, assuming positive results, we expect to be in a position to file an NDA in the 2017 timeframe."
The multi-centre trial has been designed to assess the long-term safety and tolerability of patisiran in ATTR amyloidosis patients with FAP who were previously enrolled in the Apollo Phase III study.
In the study, the patients will receive patisiran at a dose of 0.3mg/kg every three weeks for up to the earlier of two years.
Its primary objective is to assess the long-term safety and efficacy of patisiran administration and it also measures multiple clinical endpoints, including Neuropathy Impairment Score at baseline and every 12 months thereafter.
In addition, the company is carrying out a Phase II Ole study, where patients previously treated in a Phase II study are receiving open-label patisiran at a dose of 0.3mg/kg every three weeks.
In January 2014, Alnylam partnered with Sanofi company Genzyme to develop and commercialise RNAi therapeutics across the globe.
Under the deal, Alnylam will take the responsibility of patisiran in North America and Western Europe, while Genzyme will cover in the rest of the world.