Phase III data on Hemgenix published in NEJM

23 February 2023

Today, Dutch gene therapy company uniQure (Nasdaq: QURE) announced that the prestigious New England Journal of Medicine (NEJM) has published data from the pivotal Phase III HOPE-B study evaluating the efficacy, durability and safety of Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy approved for the treatment of adults with hemophilia B.

Earlier this week, the European Commission approved Hemgenix, dubbed the world’s costly drug, and out-licensed globally to Australian biotech CSL Limited (ASX: CSL), which follows the US approval in November 2022. The approval applications were supported by the HOPE-B study.

Results from the HOPE-B trial, the largest gene therapy study in hemophilia B to-date, showed that Hemgenix is superior to routine factor IX (FIX) prophylaxis in Annualized Bleeding Rate (ABR), FIX activity, FIX therapy consumption, FIX infusion rate, and spontaneous and joint bleeding ABR.

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