Experimental Drug for Metachromatic Leukodystrophy Gets Rare Pediatric Disease Designation

According to a story from EPM Magazine, the pharmaceutical company Orchard Therapeutics received Rare Pediatric Disease Designation for its gene therapy drug candidate OTL-200. The therapy is in development for the treatment of metachromatic leukodystrophy. This is the fourth candidate from Orchard to receive the designation.

Metachromatic leukodystrophy (MLD) is a type of lysosomal storage disease which affects the development and growth of myelin, an essential component of the immune system which acts as an insulating cover for nerve cells throughout the body. In metachromatic leukodystrophy, cerebroside sulfate builds up in body tissue. This genetically inherited disease is caused by a deficiency of arylsulfatase A enzyme. There is a late infantile, juvenile, and adult form, with the earliest onset forms being the most severe; the most common late infantile type kills patients by age five. Symptoms include muscle rigidity, weakness, and degeneration, developmental delays, blindness, dementia, paralysis, and convulsions. Most treatments involve managing disease symptoms, although patients that are pre-symptomatic can slow disease progression with a bone marrow transplant operation. To learn more about metachromatic leukodystrophy, click here.

The Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) is reserved for therapies that are in development to treat diseases that meet certain criteria. The primary criteria is that the disease must be serious and life threatening. In addition, it must most affect individuals aged 18 or older. The disease must also be considered rare; under the law, any illness that affects 200,000 people in the country or less can qualify as rare. The company whose product receives the designation is given a priority review voucher that the can choose to sell or transfer.

MLD definitely falls into this category as a rare and deadly disease that mostly affects children. So far, OTL-200 has displayed the capability to potentially halt the progression of both mental and physical symptoms associated with MLD. Orchard hopes to file for marketing authorization for the treatment in 2019 if trials proceed as planned. Its approval would make it one of the first treatments approved for this severe and lethal disease. Hopefully, this investigational treatment will represent a major breakthrough for the treatment of MLD.


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