The first drug was approved Friday for a rare genetic disorder that stunts growth and causes rapid aging in children, after studies showed it can extend their lives.
Kids with the genetic disorder progeria typically die in their early teens, usually from heart disease. But in testing, children taking the drug Zokinvy lived 2 1/2 years longer on average.
The U.S. Food and Drug Administration approved the capsules for progeria and a related condition.
Research on the treatment was mainly funded by the Progeria Research Foundation in Peabody, Massachusetts, with help from drug developer Eiger BioPharmaceuticals.
“This is just the first. We’ll find more and better treatments,” said Dr. Leslie Gordon, the foundation’s medical director.
Gordon, a pediatric disease researcher at Hasbro Children’s Hospital in Providence, Rhode Island, created the foundation in 1999 with her sister and husband, soon after their son Sam was diagnosed. He died in 2014 at age 17.
Just an estimated 400 people worldwide have progeria or its related condition, including 20 in the U.S. The disorder causes stunted growth, stiff joints, hair loss and aged-looking skin. Children with the disease suffer strokes and hardening of heart arteries, and die at 14 1/2 on average.
The disorder is not inherited but due to a chance gene mutation that causes a damaging buildup in cells of a protein called progerin, for which the disorder is named. The drug blocks production and accumulation of the protein, slowing its damage and the premature aging.…