Atidarsagene autotemcel (Lenmeldy) is approved for presymptomatic late infantile, presymptomatic early juvenile, or early symptomatic early juvenile MLD.
Atidarsagene autotemcel was granted priority review and had orphan drug, rare pediatric disease, and regenerative medicine advanced therapy designations.
MLD affects an estimated 1 in every 40,000 individuals in the United States. There is no cure. Treatment typically focuses on supportive care and symptom management. In children with MLD, treatment with Lenmeldy significantly reduced the risk for severe motor impairment or death compared with untreated children .
In addition, children with presymptomatic early juvenile and early symptomatic early juvenile MLD showed slowing of motor and cognitive disease.
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