Zokinvy, the first drug to improve the life expectancy of patients

Progeria is a disease rare which affects about one in 4-8 million births, according to Inserm estimates. Also known as Hutchinson-Gilford syndrome, it is characterized by the abnormal aging of cells that occurs in the first few months of life.

Children suffering 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 on average 12-13 years.

To increase 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 this type

Led by the American Progeria Research Foundation (Peabody, Massachusetts, United States), this research is the first to present the results that have developed a drug for the treatment of this disease. The treatment in question, called Zokinvy, comes in the form of capsules.

“This is just the first. We will find other even more effective treatments “says Dr. Leslie Gordon, medical director of the Progeria Research Foundation and a researcher in pediatric diseases at Hasbro Children’s Hospital (Providence, Rhode Island, USA).

Although not inherited, progeria is caused by a genetic mutation that causes a harmful buildup in cells of a protein called progerin. Zovinky would block the production and accumulation of progerin, which would have the effect of slowing down premature aging.

An extended life expectancy of two and a half years

A total of 62 children took the drug twice a day. Their results were compared to those of 81 untreated children around the world, 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 the Children’s Hospital of Boston, 22 children and young adults who have taken this drug since 2010 or earlier. Leslie Gordon sees hope in this treatment to enable 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 released but it risks being expensive given the rarity of the disease. The Californian company responsible for its development, Eiger BioPharmaceuticals, however, promises financial assistance so that all patients can benefit from it.