FDA hold for gene therapy program in Fabry disease

6 February 2023

Genetic medicines company 4D Molecular Therapeutics (Nasdaq: FDMT) has announced that its Fabry disease gene therapy program has been placed on clinical hold by the US Food and Drug Administration (FDA).

There are currently two clinical trials, one in the USA and one in Taiwan and Australia, evaluating a single intravenous administration of 4D-310 in classic or late-onset Fabry disease.

Last month, the Californian company reported three cases of atypical hemolytic uremic syndrome (aHUS) in the first six patients enrolled in the program.

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