The FDA has approved a treatment for a rare and fatal disease that causes children to age prematurely.
Called Hutchinson-Gilford progeria syndrome, the condition is triggered by a random genetic mutation that causes children to display symptoms of accelerated aging by the time they’re two-years-old. On average, patients die of a heart attack, heart failure or stroke by the time they turn 15.
On Friday, the FDA approved Zovinky, the first-ever treatment for progeria, which blocks the buildup of defective progerin. The treatment was developed by Eiger BioPharmeceuticals, who took over the treatment after it was dropped Merck. In a clinical trial of 62 patients, Zovinky increased the average time of survival by 2.5 years.
As part of the approval, the FDA granted Eiger a rare pediatric disease priority review voucher, which can be used to speed the regulatory review of another drug. Eiger intends to sell the voucher and share proceeds with the Progeria Research Foundation.
