FX.co ★ FDA Approves First Gene Therapy For Kids With Early-onset MLD

The U.S. Food and Drug Administration has given its approval to Lenmeldy (atidarsagene autotemcel), a groundbreaking gene therapy designed to treat children with early-onset metachromatic leukodystrophy (MLD). MLD is a rare, life-threatening genetic disease which causes damage to the brain and nervous system due to an enzyme deficiency. At present, no cure for MLD exists, and treatments typically centre around managing symptoms.

Lenmeldy was developed by biotechnology company, Orchard Therapeutics, which was recently purchased by Kyowa Kirin Co., a pharmaceutical and biotechnology company based in Japan. The gene therapy is marketed in Europe under the brand name Libmeldy.

This therapy is a one-time single-dose infusion constructed from the patient's own blood stem cells. These cells have been altered to include functional copies of the deficient enzyme's gene, arylsulfatase A (ARSA). Following genetic modification, the stem cells are re-introduced to the patient, where they multiply within the bone marrow. Treatments with Lenmeldy demonstrate potential to restore enzymatic function and slow or halt disease progression.

The approval of Lenmeldy is based on studies involving 37 pediatric patients with early-onset MLD. Patients who received Lenmeldy treatments showed a significant reduction in the risk of severe motor impairment or death compared to untreated children.

Orchard Therapeutics' CEO, Bobby Gaspar, expressed that the FDA's approval of Lenmeldy is the outcome of years of research and development in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

In recognition of the development of this ground-breaking treatment, Lenmeldy received both a Rare Pediatric Disease and a Regenerative Medicine Advanced Therapy designation from the FDA, as well as a Priority Review in September 2023. Furthermore, a Priority Review Voucher was issued to Orchard Therapeutics, which will be transferred to GlaxoSmithKline (GSK) as per the terms of the original licensing agreement.

In Europe, both Lenmeldy and Libmeldy have received approval from the European Commission, UK Medicines and Healthcare products Regulatory Agency, and the Swiss Agency for Therapeutic Products.