Madeleine Powys, MBBS, on Delivering Libmeldy to Children With Metachromatic Leukodystrophy

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the limited distribution model of arsa-cel in Europe.

“Because these are rare diseases, it's important that you have centers of excellence that are qualified treatment centers where there's clinical, nursing, and laboratory expertise... There's been an enormous amount of effort put into training of all the staff on the various complex steps that need to occur to make sure that the product is safely collected, manufactured and delivered into our patients.”

Atidarsagene autotemcel (arsa-cel; Orchard Therapeutics), approved under the name Libmeldy by the European Commission in 2020, is being delivered to children with metachromatic leukodystrophy (MLD) under a limited distribution model that includes 5 centers of excellence trained as qualified treatment centers(QTC) across the continent. The model was presented in a poster at the 2023 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in Orlando, Florida, February 15-19, 2023.

CGTLive spoke with Madeleine Powys, MBBS, a locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital, which is the only QTC for arsa-cel in the United Kingdom, to learn more about her experience delivering arsa-cel to children and challenges that remain. She also stressed the importance of increasing awareness of later-onset forms of MLD to enable treatment in these populations.

For more coverage of the 2023 Tandem Meetings, click here.

Wynn RF, Jones S, Calbi V, et al. Atidarsagene autotemcel, a European post-regulatory approval model for delivery of autologous hematopoietic stem cell gene therapy products via a network of qualified treatment centers (QTCs). Presented at: 2023 Tandem Meetings, February 15-18, Orlando, Florida. Poster #296
Related Videos
Olivier Danos, PhD, on Refining and Specifying AAV Gene Therapy
Paul Harmatz, MD, on Reducing GAGs in MPS Type 2 With RGX-121 Gene Therapy
Francesca Barone, MD, PhD, on Positive Biomarker, Overall Survival Data With CAN-3110 in High-Grade Glioma
Sung-Yun Pai, MD, on High T-Cell Reconstitution in X-SCID With Lentiviral Gene Therapy
Related Content
© 2023 MJH Life Sciences

All rights reserved.