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!
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* Libmeldy is the EU commercial name for the gene therapy and is a trademark of Orchard Therapeutics.