Gene Therapy Is Now First FDA-Approved Treatment for Rare & Fatal CNS Disorder

MedCity News March 18, 2024
Frank Vinluan

Orchard Therapeutics gene therapy Lenmeldy won FDA approval for treating the rare enzyme deficiency metachromatic leukodystrophy. In January, Kyowa Kirin completed its acquisition of Orchard, which now operates as a subsidiary of the Japanese drugmaker.

A genetic medicine at the heart of a $387 million Kyowa Kirin acquisition has won FDA approval, a regulatory decision that makes the one-time treatment the first approved therapy for the rare inherited disorder metachromatic leukodystrophy (MLD).

In the most severe form of MLD, babies develop normally only to rapidly lose the ability to walk, talk, and interact by the time they reach late infancy. Eventually, children with MLD deteriorate to a vegetative state, and most pass away within five years of the disease’s onset.

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