On Friday, the FDA gave approval for the first medication able to actually treat the rare disease, Hutchinson–Gilford progeria syndrome (HGPS), which causes premature aging. Prior to the development of this medication in 2007, only palliative care was available to treat the disease.
HGPS is an extremely rare disease affecting only some 400 people worldwide, the disease is caused by a chance genetic mutation that causes the buildup of a protein called progerin in the cells of the afflicted person, giving the disease its name. The disorder causes stunted growth, alopecia, and aged skin appearance. Children who have the disease develop atherosclerosis, and most die of stroke. The average life expectancy for sufferers is 14 1/2 years. The medication blocks the buildup of progerin, impeding the premature aging effect.
A clinical trial followed participants for 11 years, and the medication was found to extend life by 2 1/2 years. The oldest participant is 24, having taken the medication for 13 years.
The new medication, Zokinvy, also known as lonafarnib, is expected to be expensive due to the small number of patients but the manufacturer, Eiger, said that it will offer finance plans to make it affordable for all patients. Eiger also helped finance the research for the treatment, along with the Progeria Research Foundation.
“This is just the first. We’ll find more and better treatments,” said Dr Leslie Gordon, the foundation’s medical director.
Source: Medical Xpress
