WFY Today

You need to know about the rare genetic disease that now has a cure.

At $4.25 million per treatment, a therapy designed for kids with a rare genetic disease known as MLD has emerged as the most costly medication.

Orchard Therapeutics, the company that manufactures the life-saving treatment for children with metachromatic leukodystrophy (MLD), revealed on March 20 that the medication costs $4.25 million, making it the most expensive drug in the world.

Lenmeldy is the first medication approved by the US Food and Drug Administration (FDA) for the treatment of MLD in children. It is also the first treatment for the terrible illness that claims the lives of kids before they reach seven. Every year, over 40 babies in the US are born with ML“.

The manufacturing company said in a press release that Lenmeldy, formerly known as OTL-200, will support the management of children with early stages of mental retardation (MLD), also known as pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ).

For children in the US with early-onset MLD who previously had no treatment options outside of supportive and end-of-life care, the FDA’s approval of Lenmeldy opens up a world of new possibilities.

MLD is a rare disease that affects children and their families severely and progresses quickly. It limits their quality of life and eventually results in death.Decades of research and development in collaboration with our academic and clinical partners at the San Raffaele-Telethon Institute for Gene Therapy have culminated in this accomplishment.


Metachromatic leukodystrophy, or MLD, is a rare genetic neurometabolic illness that affects the nerve system and progresses over time.

It results in a deficiency of an essential enzyme, which accumulates toxic materials in the brain and nerves.

Loss of abilities, physical weakness, and developmental delays are among the symptoms.

MLD can be lethal and advances quickly.When given to the child at the onset of symptoms, lentemyl may be able to “stop or slow the progression” of the illness with just one therapy.

Using a similar business plan and infrastructure, the introduction of Lenmeldy in the US will build on our success in providing customized gene therapies to eligible children with MLD.

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