Cambridge based Alnylam has begun a rolling FDA filing for its RNAi drug patsiran. The experienced gene silencing professional anticipates completing the process by the end of 2017, with a goal to be granted approval in the U.S. next year.
Alnylam has started the regulatory process by filing the nonclinical and chemistry, manufacturing and controls sections of its application. That will permit the FDA to start its review of these components while Alnylam prepares its clinical data for submission. Alnylam, alongside with its partner Sanofi Genzyme, is also working to win approval for patisiran in the rest of the world, beginning with a filing in the European Union. The partners anticipate receiving a filing to European regulators near the end of 2017.
This filing signifies the start of a new phase in the company’s 15-year ride to get an RNAi treatment to market. With phase 3 data indicating patisiran is safe and cuts symptoms of neuropathological impairment, Alnylam looks to be in a good position to win approval, while it waits for its new obstacle of commercializing an RNAi drug.
Alnylam will be focused on commercialization in the U.S., Canada and Western Europe, while Sanofi Genzyme will take the lead with the rest of the globe. The sales and marketing teams will most likely have to convince payers and prescribers that patisiran is a better value than Ionis’ rival hATTR drug, inotersen.
Patisiran defeated inotersen in the latest cross-trial comparison, though Ionis believes the route of administration and speed of onset of efficacy of its candidate could be the advantages they need to have success in the marketplace.