Alnylam's advance continues with NDA acceptance

2 February 2018

The momentum has been maintained in the march of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary (hATTR) amyloidosis, towards the US market

The US Food and Drug Administration (FDA) has accepted for filing Alnylam Pharmaceuticals’ (Nasdaq: ALNY) New Drug Application (NDA) for the treatment, which created headlines and investor excitement in September by delivering the first ever positive Phase III results for an RNAi therapeutic.

Since then patisiran has received Breakthrough Therapy designation from the FDA and been granted accelerated assessment by the European Medicines Agency, which has accepted the Marketing Authorization Application (MAA) and initiated its review of the drug.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK