UK's MHRA grants early access to Amicus' AT-GAA

8 June 2021

US biotech company Amicus Therapeutics (Nasdaq: FOLD) saw its shares edge up nearly 2% to $10.27 in early trading today, after it announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) to AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease.

This positive opinion means that eligible adults living with late-onset Pompe disease (LOPD) who have received alglucosidase alfa for at least two years can now switch and have access to AT-GAA prior to marketing authorization in the UK.

The EAMS mechanism is intended to provide individuals in the UK who live with a life threatening or seriously debilitating condition early access to innovative and unlicensed medicines for which there is not yet marketing authorization and where there is a clear unmet medical need.

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