Infusion For Rare Disease In Children Raises Questions Of Gene Therapy Access

The Washington Post (8/9, Roubein) reported London-based pharmaceutical company Orchard Therapeutics’ drug, Lenmeldy, is the “world’s most expensive drug,” costing $4.25 million for a “one-time infusion to stave off a rare neurodegenerative disease that can claim the lives of children by the time they turn 5.” While “the drugmaker and doctors say the treatment is most effective if given before symptoms arise,” the majority “of newborns aren’t screened for” metachromatic leukodystrophy and only five hospitals in the US “are likely to offer the infusion, according to the manufacturer, forcing many patients to travel far from home.” The number “of single-dose gene therapies approved by the FDA” has grown from zero to 18 in under seven years, “a number that analysts estimate will nearly quintuple by 2032.” However, with prices for these treatments “projected to reach a total of $35 billion to $40 billion over the next decade,” policymakers and drug manufacturers face increased pressure “to find a model for making these new medications accessible and affordable.”