The FDA recently approved the gene therapy Lenmeldy for kids with an incurable disease with no previous treatments.
A lifesaving gene therapy for children born with a rare and debilitating disease has just been approved by the U.S. Food and Drug Administration. The catch? Its wholesale cost has been set at $4.25 million, making it the most expensive medicine in the world.
The FDA and Orchard Therapeutics estimate 1 in 40,000 Americans are affected by the rapidly progressive disease each year, meaning fewer than 40 children face the poor prognosis annually. The"one-time, individualized single-dose infusion," as the FDA describes it, works by adding functional copies of the ARSA gene to a patient's own blood stem cells. After a high dose of chemotherapy, a doctor would collect the cells to genetically add the vital enzyme to their cells before transplanting the modified cells back into the patient, the FDA says.
A trial in which 37 pediatric patients with early-onset MLD received a one-time administration of Lenmeldy left each one with a significant reduction in severe motor impairment or death compared to untreated children, the FDA said. Orchard Therapeutics reported each child was alive at the age of 6 compared to only 58% of the untreated group, and at the age of 5, 71% were able to walk without help and 85% had normal speech and performance IQ scores — a finding not reported in the control group.
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