MLD Gene Therapy Approved in Europe

Gene therapy for early-onset MLD patients has been approved in all 27 EU member states as well as the UK, Iceland, Liechtenstein, and Norway for early-onset (late infantile and early-juvenile) patients.

Congratulations to the San Raffaele, TIGET, and Orchard Therapeutics teams, the clinical trial families, and the entire MLD community on this significant milestone! MLD Foundation first started working with the San Raffaele/TIGET researchers in 2005 and has been fully engaged and actively supporting the project throughout its pre-clinical phase and subsequently nearly a decade of human trials. The results in the target patient population have been outstanding. We are excited to continue on the journey toward approval & access across the EU … and throughout the world.

Dean Suhr, President & Co-Founder, MLD Foundation

Following the receipt of a European Medicines Agency (EMA) positive opinion last November recommending full marketing authorization for Libmeldy™, the gene therapy becomes the first gene therapy for eligible patients with early-onset MLD, and MLD’s first commercial therapy.

LATE-INFANTILE & EARLY-JUVENILE FORMS

Presymptomatic late-infantile and early-juvenile forms of MLD are included in the approval. The approval also includes early-juveniles with early clinical manifestations (symptoms) who are still able to walk independently and have not started to decline cognitively.

OTHER FORMS of MLD … and ACCESS IN OTHER COUNTRIES, INCLUDING THE USA

Orchard has a clinical trial underway studying Libmeldy in the late juvenile form of MLD, however, it should be noted the late juvenile and adult forms are not covered by this commercialization approval.

Patients outside the EU and with later-onset forms of MLD are able to request Orchard for access to Libmeldy via Orchard’s Pre-Approval Access/Compassionate Use programs. Orchard reviews each request on a case by case basis. MLD Foundation will help you advocate for access for your loved loved ones.

THERE IS STILL MUCH LEFT TO DO

Access and reimbursement … EMA and EC approval is a huge milestone, but it is not the finish line. MLD Foundation will continue to work directly with Orchard, EU partners, and country officials as each EU member country undertakes its own Libmeldy price and reimbursement review to make the therapy formally made available to its citizens.

FDA (USA) … MLD Foundation has been in communication with the FDA and is in full support of Orchard’s IND activities that we expect will lead to a BLA request for review and eventual approval in the US.

Newborn screening … MLD Foundation is leading an MLD newborn screening development, validation, and implementation effort to identify patients pre-symptomatically. We need your participation to make this effort successful. Click here to learn more and join us!

Subscribe to this blog to be kept updated on gene therapy and other therapeutic advances, and ongoing efforts to improve other aspects of MLD quality of life.

Orchard Therapeutics Press Release

* Libmeldy is the EU commercial name for the gene therapy and is a trademark of Orchard Therapeutics.
* The image on this page is a screen capture of Orchard Therapeutic’s homepage featuring one of the first MLD gene therapy patients.

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