The European Medicines Agency EMA announces the green light for the first treatment for children suffering from Hutchinson-Gilford progeria, the so-called ‘Benjamin Button syndrome’, or progeroid laminopathies. The EU regulatory body’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorization for Zokinvy (lonafarnib), to be administered in exceptional circumstances to children aged one year and over with diagnosis of genetically confirmed pathology. The therapy is formulated in hard capsules which are taken twice a day.
Hutchinson-Gilford progeria is an ultra-rare disease (the incidence is one in 4 million births, the prevalence of one patient in 20 million people) and multisystemic, manifested by super-premature aging that produces signs in the appearance and functionality of organs and systems; most sufferers die of severe cardiovascular complications at an average age of about 14 years. Progeroid laminopathies are even rarer.
Zokinvy is a specific inhibitor of the enzyme farnesyltransferase, explains Ema. The drug has been shown to prevent the formation of aberrant progerin and progerin-like proteins in cells, thus promoting the maintenance of their integrity and function. In addition, over time continued therapy reduces progerin levels and treated patients survive longer, on average about 6 months. In clinical trials, most patients on therapy experienced moderate or severe adverse events during the first 4-6 months of treatment. The most commonly reported side effects were vomiting, nausea, diarrhea, fatigue, upper respiratory tract infection, decreased appetite, and headache.