Following the US Food and Drug Administration approval last Friday of its Lenmeldy (atidarsagene autotemcel), Orchard Therapeutics today announced launch plans for the gene therapy.
The drug is for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile, (PSEJ), or early symptomatic early juvenile (ESEJ) - collectively referred to as early-onset - metachromatic leukodystrophy (MLD).
Orchard, which was recently acquired by Japan’s Kyowa Kirin (TYO: 4151), has set the wholesale acquisition cost (WAC) of Lenmeldy in the USA at $4.25 million which is reflective of the value the therapy may deliver to eligible patients and their families, as well the potential long-term impact treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers, and life opportunities for patients.
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