The rate of FDA approvals of gene therapies is picking up….. at a pace not too dissimilar to the bevy of biosimilars we’ve witnessed in recent years. The newest gene therapy is Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics. Lenmeldy is the first FDA-approved gene therapy that is indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
MLD is caused by a mutation on the ARSA gene, resulting in a deficiency of the enzyme arylsulfatase A (ARSA). This deficiency causes sulfatides to build up in cells and damage the central and peripheral nervous systems. As a result, patients experience a loss of motor and cognitive function and early death. MLD, which has no cure, is estimated to affect one in 40,000 individuals in the United States.
Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene. The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene. The modified stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow. The appropriate dose is calculated based on the child’s weight at time of Lenmeldy infusion using the instructions provided by the manufacturer. The processed dose is shipped to the treatment center in liquid nitrogen at less than -130°C (-202°F).
Lenmeldy now holds the austere position of being the most expensive therapy sold in the US. Orchard set the wholesale acquisition cost for Lenmeldy at $4.25 million.
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