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.

We’re thankful – even in 2020!

We heard earlier this week that nearly 4 in 5 people are already looking forward to putting 2020 behind us. The pandemic (family safety, work, school, loss of loved ones, individual and community response, social isolation, etc.), US elections, income, health, … it is affected every one of us, and it’s overwhelming.

But we are still thankful…

… not for MLD, not for loss, but for all of you – our friends, family, the MLD Family™, the greater MLD community, the MLD researchers and clinicians, regulators, policymakers, and the rare disease ecosystem who supports us.

The MLD Family™ …

… is supporting each other. There is advice, compassion, and the sharing of excess or needed supplies. We can’t meet in person, but we’re connecting with regular men’s, women’s, and now couple’s calls and so much more as we adapt to new ways of connecting.

MLD Research is progressing …

Orchard Therapeutics has achieved EMA/CHMP recommendation for approval of gene therapy in Europe. They have also filed an IND with the US FDA, a key stepping stone formalizing communications with the FDA as they move one step closer to US review and approval.

Takeda continues its ERT clinical trial. Homology Medicine continues to make pre-clinical progress with their gene therapy. And we are working with several other biopharma companies, too!

MLD Newborn Screening …

We have to diagnose children earlier in order for them to have good viable life-saving therapy options!

The MLD Newborn Screening program is making great progress. New York is right on the cusp of launching an MLD pilot study into parts of their state. The MD NBS Expert Advisory Group is meeting monthly. Many of you are participating with the Working Focus Groups – you can still join us for our December meetings!

MLD Cinical Care Continues to Improve …

We are blessed to have many wonderful and knowledgable MLD clinical researchers … we still need more, but those who do support our community are open, supportive, and often go the extra mile as they care for our loved ones in person or remotely. We worry about their health as they are called into care situations where additional COVID resources are necessary – let’s all keep doing our part to keep these COVID care needs minimized and our clinicians safe!

In 2020 we started to lay the foundation for creating an MLD Standard of Care so we can all more easily get approval and reimbursement for better, more consistent, and more appropriate MLD clinical care.

Let’s keep connected …

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

Gene Therapy Recommended for Full EU EC Marketing Authorization Review

Gene therapy for early-onset MLD patients passed a huge milestone yesterday when the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy, by the European Commission (EC), which has the authority to grant marketing authorization for Libmeldy* in the European Union (EU). The EC is anticipated to make a final decision by the end of 2020. If approved, Libmeldy™ would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

LATE-INFANTILE & EARLY-JUVENILE FORMS RECOMMENDED

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

THERE IS MUCH STILL LEFT TO DO

After the EC marketing authorization is approved, each EU member country will have to do its own Libmeldy price and reimbursement review before the therapy is available to its citizens.

MLD Foundation is leading an MLD newborn screening development, validation, and implementation effort to identify patients pre-symptomatically. We need family 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.