Shares of US biotech firm Magenta Therapeutics (Nasdaq: MGTA) edged up more than 2% to $9.97, after it announced that the US Food and Drug Administration granted Regenerative Medicine Advance Therapy (RMAT) designation for MGTA-456, a one-time cell therapy for the treatment of multiple inherited metabolic disorders.
“This RMAT designation was based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative, life-saving potential of MGTA-456 for children suffering from inherited metabolic disorders,” said Dr John Davis, chief medical officer of Magenta, adding: “We look forward to collaborating closely with the FDA as we seek to rapidly advance MGTA-456 through the ongoing Phase II study and into potential pivotal studies in 2020.”
Formerly dubbed HSC835, the MGTA-456 program was in-licensed from Swiss pharma giant Novartis (NOVN: VX) in May 2017.
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 2024 | Headless Content Management with Blaze