BLA Submission Gives Libmeldy a Shot at US Review for Metachromatic Leukodystrophy


The gene-edited cell therapy OTL-200 was approved in Europe in 2020.

Orchard Therapeutics has completed its rolling biologics license application (BLA) for priority review of OTL-200 (atidarsagene autotemcel) for treating children with early-onset metachromatic leukodystrophy (MLD).1 If accepted, the FDA would make a decision on the therapy in the first half of 2024.

“With the completion of the rolling BLA submission for OTL-200 to the FDA, we are now one significant step closer to bringing this important therapy to families in the US affected by MLD who currently have no treatment options beyond supportive care,” Bobby Gaspar, MD, PhD, chief executive officer, Orchard, said in a statement.1 “We look forward to working with the agency throughout the filing and review process and expect to hear from the FDA regarding acceptance of the BLA in the third quarter of this year.”

OTL-200 was approved in Europe in 2020 for treating MLD under the name Libmeldy.2 It is available under a limited distribution model that includes 5 centers of excellence trained as qualified treatment centers across the continent. The FDA has previously granted the therapy Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations.

Orchard also gave several business updates while announcing the BLA submission, including new reimbursement agreements in 4 additional European countries, most recently, Norway.1 The company is continuing its newborn screening studies and efforts, which have recently suggested an incidence rate of closer to one in 50,000 live births compared to prior estimates in medical literature of one in 100,000. Screening efforts have made progress in both Germany and Illinois, which recently passed the Newborn Metabolic Screening Act.

WATCH NOW: Madeleine Powys, MBBS, on Lessons Learned With Libmeldy

“We are proud to support newborn screening research for MLD to generate the data necessary to enable the implementation of universal MLD screening, which will help ensure timely diagnosis and treatment referral so children and their families can be offered the opportunity for the best possible outcomes. The data generated thus far from these studies suggests a significantly higher incidence than was previously estimated in the literature and may lead to a greater commercial opportunity for Libmeldy,” Braden Parker, chief commercial officer, Orchard, added to the statement.1 “We remain on track to meet our goal of year-over-year revenue growth in 2023, and with the completion of our rolling BLA submission for OTL-200 in MLD, we are ramping up our US prelaunch activities.”

Recent long-term data of up to 11 years' follow-up from 2 clinical trials (NCT01560182, NCT03392987) conducted at San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, and expanded access programsdemonstrated a continued favorable risk/benefit profile in patients with pre-symptomatic late-infantile and early-juvenile (EJ), and early-symptomatic EJ MLD.2

The data, which were presented at the WORLDSymposium 2023, held February 22-26, in Orlando, Florida, by Francesca Fumagalli, MD, neurologist, IRCCS Ospedale San Raffaele, showed that all patients achieved hematological recovery, stable engraftment, and restoration of arylsulfatase A enzyme to normal or supranormal levels by 3 months in peripheral blood mononuclear cells and by 3 to 12 months in cerebrospinal fluid.

1. Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA. News release. Orchard Therapeutics. August 3, 2023.
2. Fumagalli F, Calbi V, De Mattia F, et al. Long-term clinical outcomes of atidarsageneautotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up. Presented at: WORLDSymposium2023, February 22-28; Orlando, Florida. Abstract #125
Related Videos
Jeffrey Chamberlain, PhD
Robert J. Hopkin, MD
Alan Beggs, PhD
Paul Harmatz, MD
Paul Harmatz, MD
Heather Lau, MD, MS, the executive director of global clinical development at Ultragenyx Pharmaceutical
Heather Lau, MD, MS, the executive director of global clinical development at Ultragenyx Pharmaceutical
Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes
Paul Harmatz, MD, on Assessing D2S6 in Trials for MPS Type 2
© 2024 MJH Life Sciences

All rights reserved.