Orchard Therapeutics’ BLA for Metachromatic Leukodystrophy Cell Therapy Arsa-Cel Accepted With Priority Review
The PDUFA data has been set for March 18, 2024.
Orchard Therapeutics’ biologics license application (BLA) for atidarsagene autotemcel (arsa-cel, also referred to as OTL-200 and approved as Libmeldy in the European Union, UK, Iceland, Liechtenstein, and Norway), a gene-edited cell therapy being evaluated for the treatment of metachromatic leukodystrophy (MLD), has been accepted by the FDA with priority review.1,2 The Prescription Drug User Fee Act (PDUFA) date is set for March 18, 2024.
“Today is another significant step forward for patients and families in the US 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,” Bobby Gaspar, MD, PhD, the cofounder and chief executive officer of Orchard Therapeutics, said in a statement.1 “We look forward to collaborating with the FDA throughout the review and evaluation of our application. Due to the nature of the disease and the urgency to treat children affected by MLD, we are working diligently in parallel to prepare for a potential launch in 2024 and ensure OTL-200 will be available to patients in the US as quickly as possible.”
The company
“MLD is a devastating and ultimately fatal disease for which there are no alternative treatment options beyond supportive care,” Leslie Meltzer, PhD, the chief medical officer of Orchard Therapeutics, said in an August 2023 statement.2 “These data, which now encompass more than a cumulative 250 years of patient experience, continue to show sustained preservation of cognitive function and motor development in most patients compared to disease natural history consistent with previously published results. Moreover, results from the updated integrated analysis presented at SSIEM are key components of the clinical package in the OTL-200 BLA we recently submitted to the FDA.”
Orchard
REFERENCES
1. Orchard Therapeutics announces acceptance of biologics license application for OTL-200 in MLD and receives priority review. News release. Orchard Therapeutics. September 18, 2023. Accessed September 18, 2023. https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-acceptance-biologics-license
2. Orchard Therapeutics announces presentation of data comprising the clinical package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023. News release. Orchard Therapeutics. August 31, 2023. Accessed September 1, 2023. https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-presentation-data-comprising
3. Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA. News release. Orchard Therapeutics. August 3, 2023. Accessed September 1, 2023. https://finance.yahoo.com/news/orchard-therapeutics-completes-submission-biologics-110000497.html
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Related Articles
- Top News in Lymphoma Cell Therapy for World Lymphoma Awareness Day 2025
September 15th 2025
- Duchenne Action Month 2025: Looking Back at News and Expert Insights
September 14th 2025
- CGTLive®’s Weekly Rewind – September 12, 2025
September 12th 2025