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The first drug was approved on Friday for a rare genetic condition that slows growth and causes children to age rapidly, after studies have shown it can extend their lives.
Children with the genetic progeria disorder usually die in their early teens, usually from heart disease. But during tests, children taking the drug Zokinvy lived 2.5 years longer on average.
The United States Food and Drug Administration has approved the capsules for progeria and a related condition.
Treatment research was funded primarily by the Progeria Research Foundation in Peabody, Massachusetts, with help from drug developer Eiger BioPharmaceuticals.
“This is only the first. We will find more and better treatments, ”said Dr. Leslie Gordon, medical director of the foundation.
Gordon, a pediatric disease researcher at Hasbro Children’s Hospital in Providence, Rhode Island, established the foundation in 1999 with her sister and husband, shortly after their son Sam was diagnosed. He died in 2014 in 17 years.
About 400 people worldwide suffer from progeria or its associated disease, including 20 in the United States. Children with the disease suffer from strokes and hardening of the 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 the production and accumulation of the protein, slowing its damage and premature aging.
Until testing began in 2007, doctors could only try to alleviate certain symptoms.
Meghan Waldron of Deerfield, Massachusetts, was diagnosed with progeria at the age of 2. She was not growing or gaining weight and her hair was falling out. She was one of the first children to receive the drug.
“Soon,” she said, “there were obvious improvements.”
She started to grow a little more – she is now 3 feet 7 inches tall – and tests have shown a slowing in the hardening of her arteries.
Waldron, 19, traveled to Europe on her own last year after graduating from high school, where she competed in track and field and cross country.
“My physical health is pretty good,” aside from some joint stiffness, said Waldron, a sophomore creative writing student at Emerson College in Boston. “It’s just something I live with.”
She is still taking the drug as part of a long-term follow-up study.
“I’m so excited” by his approval, she said.
The FDA’s action was based on two studies in which a total of 62 children took the drug twice a day. Their results were compared to 81 untreated children worldwide, compared by age and other characteristics.
Participants were followed for up to 11 years, and those who took the drug lived 2.5 years longer on average.
In all, four studies of the drug were conducted at Children’s Hospital in Boston, with 22 children and young adults taking the drug since 2010 or earlier. The oldest is 24 and has been taking it for 13 years.
Eiger, a small drug developer from Palo Alto, Calif., Is not yet revealing the price of Zokinvy, also known as lonafarnib, but it will be expensive because there are so few patients. Eiger will offer financial assistance so that all patients can get it.
The most common side effects of Zokinvy were vomiting, diarrhea, nausea, abdominal pain, and fatigue.
Gordon of the foundation worked with the director of the National Institutes of Health, Dr Francis Collins, on laboratory research that uncovered the genetic cause of progeria in 2003.
She said the “upcoming pike” research could potentially give patients “a longer life, a stronger heart, and a cure.”
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Follow Linda A. Johnson on Twitter: @LindaJ_onPharma
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The Associated Press’s Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education. The AP is solely responsible for all content.
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