PRIME status for ProQR's sepofarsen

29 July 2019

New York-traded shares in ProQR Therapeutics (Nasdaq: PRQR) looked set to open around 6% up on Monday with news that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) status to a drug in development for Leber’s congenital amaurosis 10 (LCA10).

ProQR is developing RNA medicines for the treatment of severe genetic diseases, with sepofarsen being developed to target the p.Cys998X mutation in the CEP290 gene in LCA10, the most common cause of blindness due to genetic disease in children.

The Dutch biotech firm sees the European Union as an important market for this medicine, and the company can take confidence from the fact that an RNA-interfering treatment has already been approved in Europe, in Alnylam Pharmaceuticals’ (Nasdaq: ALNY) Onpattro (patisiran).

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