FDA Filing Completed for MLD Gene Therapy

Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe.  Their “rolling-BLA” filings started last quarter.

The BLA is a “Biologics License Application“, the process used to get an FDA review of their gene therapy.  If approved, Accelerated Approval would reduce the FDA’s review time to six-months. This could result in a US approval in the first half of 2024.

This is a major milestone for a therapy that has demonstrated remarkable results, especially for pre-symptomatic patients, most of who are living very normal lives.

In parallel with supporting the gene therapy development and approval, MLD Foundation has been working for over a decade on newborn screening so  patients can be identified when pre-symptomatic, a genotype-phenotype research project so patients identified at birth can be referred to the proper therapy at the proper time, and on access & reimbursement so patients can get to the therapy sites and the therapies can be paid for.