Orchard gene therapy wins first FDA nod for rare pediatric disease

March 19, 2024

The U.S. FDA has approved Orchard Therapeutics' gene therapy, Lenmeldy, for the treatment of children with several types of early-onset metachromatic leukodystrophy (MLD).

Lenmeldy (atidarsagene autotemcel) was approved for children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile MLD — all of which previously had no treatment options beyond supportive and end-of-life care. MLD is a rare and life-threatening genetic disease of the body’s metabolic system. In its most severe form, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact.

According to Orchard, Lenmeldy aims to correct the underlying genetic cause of MLD by inserting one or more functional copies of the human ARSA gene ex vivo into the genome of a patient’s own hematopoietic stem cells using a lentiviral vector. In a single treatment, the repaired cells are infused back into the patient, where, once engrafted, they can restore enzymatic function to stop or slow disease progression.

The FDA approval is based on data from 37 pediatric patients with early-onset MLD, enrolled in two single-arm, open-label clinical studies or treated under European expanded access frameworks, who received a one-time administration of the gene therapy. 

The therapy, branded Lenmeldy, is already approved by the European Commission and UK Medicines and Healthcare products Regulatory Agency. The recent U.S. approval will give Orchard a Priority Review Voucher, which it has agreed to transfer to GSK through a previous licensing agreement.

The news comes just months after Japan-based global specialty pharmaceutical company, Kyowa Kirin, completed its $477.6 million acquisition of Orchard. The company is now a wholly-owned subsidiary of Kyowa Kirin.