Historic hemophilia nod paves way for world's most costly medicine

23 November 2022

In a landmark regulatory first, Dutch gene therapy company uniQure (Nasdaq: QURE) has secured US approval for its one-time hemophilia B treatment, Hemgenix (etranacogene dezaparvovec-drlb).

Further development and commercialization will be taken forward by licensor CSL Behring, part of Australia's CSL Limited (ASX: CSL), after the firm secured global rights at a potential cost of over $2 billion.

The US Food and Drug Administration granted approval for the treatment of certain adults with hemophilia B, including those who currently use factor IX prophylaxis therapy.

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