Grandparents
Orchard Therapeutics (ORTX), recently acquired by Kyowa Kirin (OTCPK:KYKOY), has received U.S. approval for its Lenmeldy gene therapy as the first FDA-approved treatment for early-onset metachromatic leukodystrophy (MLD), a rare metabolic disease.
Formerly known as OTL-200, Lenmeldy is designed to add copies of the human arylsulfatase-A (ARSA) gene, mutations of which lead to MLD.
According to FDA label information, the treatment will be indicated in the United States for children with MLD during the pre-symptomatic and early symptomatic stages, who have not previously received disease-specific therapies.
“The FDA approval of Lenmeldy opens up tremendous new possibilities for children in the United States with early-onset MLD who previously had no treatment options beyond supportive and end-of-life care,” noted Bobby Gaspar, CEO of Orchard.
Orchard (ORTX) plans to announce Lenmeldy market launch details this week. The approval came weeks after Japanese drugmaker Kyowa Kirin (OTCPK:KYKOF) acquired the British company in January for $387.4 million.