Orchard's gene therapy a step closer to US approval
Gene therapy company Orchard Therapeutics (Nasdaq: ORTX) has announced that the US Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review.
The FDA has set a Prescription Drug User Fee Act goal date of March 18, 2024. If approved, OTL-200 would be the first and only treatment in the USA for early-onset MLD.
"We are working diligently in parallel to prepare for a potential launch in 2024"Bobby Gaspar, co-founder and chief executive of Orchard, said: “Today is another significant step forward for patients and families in the USA impacted by this devastating and cruel disease who for too long have dealt with the unimaginable burden of going through the diagnostic odyssey, being told there were no treatments beyond supportive care, and then having to watch their child slip away.
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
Free
7 day trial access
- 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
- 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
More on this story...
Today's issue
Company Spotlight
More Features in Biotechnology
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze