Washington, March 19 (QNA) - The US Food and Drug Administration (FDA) announced the approval of gene therapy, provided by Orchard Therapeutics, for children suffering from metachromatic leukodystrophy (MLD), making it the first approved treatment in the United States for this rare hereditary disease.

The administration agreed to provide this one-time, individualized single-dose infusion, branded as Lenmeldy in the United States, to children at certain stages of the diseases development.

The safety and effectiveness of Lenmeldy was assessed based on data from 37 children who received the drug in two open-lable clinical trials, whose results showed that the treatment significantly reduced the risk of severe motor impairment or death compared with untreated children.

Data also flagged potential risk of blood cancer associated with the treatment, along with formation of blood clots or a type of swelling of brain tissues.

MLD affects one in every 40,000 individuals in the United States. It is a rare genetic disorder that affects children and causes the accumulation of fatty substances in cells, especially in the brain, spinal cord, and peripheral nerves, according to the FDA. (QNA)