Shares in Amicus Therapeutics dropped 32.5% to $6.42 on February 27, after the US drug developer suspended enrollment into a Phase II trial evaluating AT2220 (1-deoxynojirimycin HCI) for Pompe Disease on side effect issues.
The Food and Drug Administration has placed the drug on clinical hold. After evaluation of available data, the company says it plans to work closely with the agency and, if appropriate, will amend the Phase II protocol with the objective of restarting the trial as soon as possible.
Two patients enrolled in the trial experienced self-reported adverse events and subsequently withdrew from the study. The events were categorized by the site investigator as serious and probably related to treatment with AT2220. After initial evaluation of the cases, Amicus suspended patient enrollment and, in a subsequent discussion with the FDA, the trial was placed on clinical hold status pending further evaluation of the data.
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.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
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
Copyright © The Pharma Letter 2025 | Headless Content Management with Blaze