FDA Approves Lenmeldy for Children with Metachromatic Leukodystrophy
Orchard Therapeutics/Kyowa Kirin has announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™, formerly known as OTL-200, for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) collectively referred to as early-onset—metachromatic leukodystrophy (MLD).
Metachromatic leukodystrophy is a rare and severe genetic condition that affects the brain and nervous system. Metachromatic leukodystrophy is caused by a faulty gene, which means that the body does not make enough of an enzyme called arylsulfatase A (ARSA). That lack of ARSA causes fatty substances called sulfatides to build up in the brain and nerves, leading to problems with movement and thinking abilities, severe spasticity, seizures, and patients gradually losing the ability to move, talk, swallow, eat, and see.
Lenmeldy is a gene therapy used to treat of types of metachromatic leukodystrophy in children. Lenmeldy is made using the patient’s own stem cells that have been modified so that the body can make the ARSA enzyme to help stop the progression of MLD. Lenmeldy is given as a one-time infusion.
