Nanolek continues to expand its orphan drug portfolio. As part of its ongoing collaboration with the South Korean biopharmaceutical company GC Biopharma, Nanolek is set to expand its portfolio to include an innovative treatment for Hunter syndrome (mucopolysaccharidosis type II), known as Hunterase Neuro. The initial shipment of this drug, aimed at treating the first patients, is scheduled for November of this year.

In June, the Nanolek delegation, including Artyom An, Deputy General Director for Strategic Development, and Dmitry Dvinin, Product Manager, visited Okayama Medical Hospital in Japan. Dr. Furuojo, Chief Physician, Pediatrics Department, conducted a master class on administering the Hunterase Neuro injection. During the visit, doctors shared clinical data demonstrating the safety and efficacy of the drug.

Nanolek representatives participated in the Hunterase L.I.N.K. Day, organized by GC Biopharma. This event brought together doctors from Russia, Japan, and Colombia to share their experiences in treating Hunter syndrome. They discussed data from the Hunterase Neuro clinical trial, the drug usage experiences, and the implantation procedure for the Ommaya reservoir. This reservoir, which is used to deliver a small volume but highly concentrated enzyme directly into the patient’s brain can be used for a long-term period due to its low invasiveness. In addition, microbiological infections due to the use of drug was not observed, which attributed to the short duration of the injections.

The collaboration between Nanolek and GC Biopharma began in 2015 and led to the introduction of the drug Hunterase in Russia in 2018. Developed by South Korean biotechnologists, Hunterase is used for intravenous administration as part of enzyme replacement therapy for Hunter syndrome. The innovative drug Hunterase Neuro is administered intraventricularly—directly into the brain's ventricles—using an Ommaya reservoir. Scientists believe this method increases the effectiveness of the therapy.

For reference: Hunter syndrome, also known as mucopolysaccharidosis type II, is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This deficiency leads to an accumulation of glycosaminoglycans in the body, resulting in damage to various organs and tissues.