$4.25 million gene therapy for kids becomes world's priciest drug
Newsy
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.
Orchard Therapeutics announced the hefty price for Lenmeldy Wednesday, two days after the FDA approved the therapy as the only treatment for kids with metachromatic leukodystrophy, or MLD.
MLD is an incurable genetic disorder caused by a certain gene mutation that creates a deficiency of the enzyme arylsulfatase A (ARSA), according to the National Institutes of Health. This leads to a toxic buildup of fatty substances called sulfatides in cells, which gradually destroys the fatty covering around nerve fibers, called the myelin sheath, the NIH says. This irreversibly damages the nervous system, leading to loss of function and early death, per the NIH and FDA.
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.
Most cases are categorized as late infantile MLD, meaning symptoms begin between 6 months and 2 years of age, according to the Children's Hospital of Pittsburgh. The majority of these children die by the age of 5, the NIH says.
