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

Video

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.

REFERENCE
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
Recent Videos
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
© 2025 MJH Life Sciences

All rights reserved.