30/07/2026
The Russian Ministry of Health has granted marketing authorisation to GENERIUM for Clotilia® (INN: verenafusp alfa) — an innovative original drug for the treatment of Mucopolysaccharidosis Type II (Hunter syndrome), a severe and progressive rare genetic disease.
Hunter syndrome is caused by a deficiency of the enzyme iduronate-2-sulfatase, which leads to the accumulation of toxic sugar compounds — glycosaminoglycans (GAGs) — in the cells of virtually all organs, including the brain. The disease affects primarily children and, in its severe form, causes progressive damage to the nervous system.
Verenafusp alfa is a recombinant modified enzyme covalently bound to the Fab fragment of a monoclonal antibody against the human insulin receptor. The Fab fragment acts as a molecular transporter, binding to insulin receptors on the cells of the blood-brain barrier and triggering receptor-mediated transcytosis — a natural mechanism that guides the therapeutic molecule across the barrier and into the brain.
Once delivered, the enzyme breaks down accumulated GAGs in nerve cells, halting the pathological process. The insulin receptor as a delivery target represents a unique, patented technological platform.
Clotilia® is indicated for adults and children over 6 years of age. It will be manufactured by GENERIUM on a full-cycle basis in Russia and will become available to patients in the near future.
