The Zokinvy has been developed for more than ten years in the United States and gives hope to children with progeria. According to the results obtained during the research, this treatment, recently approved by the Food and Drug Administration, would extend the life expectancy of patients by an average of two and a half years.
-1606051215.jpg "Progeria: Zokinvy, the first drug to improve patient life expectancy")
- A new treatment against Progeria, this rare genetic disease that accelerates aging, has been approved in the United States.
- This drug, Zokinvy, saves two and a half years of life expectancy for people with this disease.
Progeria is a disease rare which concerns approximately one birth in 4 to 8 million, according to Inserm estimates. Also known as Hutchinson-Gilford syndromeit is characterized by abnormal aging of cells which occurs from the first months of life.
Children who suffer from this pathology present symptoms similar to those observed in individuals of advanced age: joint pain, cardiovascular disorders, alopecia… the life expectancy linked to progeria is 12-13 years on average.
In order to increase the life expectancy and improve the condition of these patients, American researchers have just completed clinical trials for a drug approved by the Food and Drug Administration (FDA) on Friday, November 20.
First treatment of its kind
Led by the American institute Progeria Research Foundation (Peabody, Massachusetts, USA), this research is the first to present results having developed a drug to treat this disease. The treatment in question, named Zokinvy, comes in the form of capsules.
“This is only the first. We will find other treatments that are even more effective”says Dr. Leslie Gordon, medical director of the Progeria Research Foundation and pediatric disease researcher at Hasbro Children’s Hospital (Providence, Rhode Island, USA).
Although not hereditary, progeria is caused by a genetic mutation which causes cells to build up a damaging protein called progerin. Zovinky would block the production and accumulation of progerin, which would have the effect of slowing down premature aging.
Extended life expectancy of 2.5 years
A total of 62 children took the drug twice a day. Their results were compared with those of 81 untreated children worldwide, based on their age and other characteristics. Participants were followed for 11 years, and those who took the drug lived an average of two and a half years longer.
A total of four studies were conducted at Boston Children’s Hospital, with 22 children and young adults taking the drug since 2010 or earlier. Leslie Gordon sees in this treatment a hope to allow patients to have “a longer life, a stronger heart and the possibility of moving towards a cure”.
The price of Zokinvy has not yet been revealed but could be expensive, given the rarity of the disease. The Californian company in charge of its development, Eiger BioPharmaceuticals, however, promises financial assistance so that all patients can benefit from it.
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