A First in Azerbaijan: Innovative Treatment for Metabolic Diseases Successfully Implemented
- Under the leadership of Dr. Laman Alirzayeva, Head of the Metabolic Diseases Center at Referans Medical Group, pediatrician and specialist in pediatric metabolic disorders, an 8-year-old child diagnosed with Hunter syndrome (MPS II) has become the first patient in Azerbaijan to receive KLOTILIA (verenafusp alfa), an innovative therapy capable of crossing the blood–brain barrier.
- The first stage of treatment was successfully carried out at Leyla Medical Center.
- This innovative therapy was introduced to Azerbaijan with the support of the manufacturer and was provided to the patient completely free of charge. The initiative represents a significant milestone in expanding access to advanced treatment options for children living with rare metabolic disorders in Azerbaijan.
- Hunter syndrome is a rare inherited metabolic disorder caused by the deficiency of a specific enzyme. Over time, the disease affects multiple organs, including the central nervous system. Early diagnosis and timely treatment play a crucial role in improving patients’ quality of life and long-term outcomes.
- Until now, enzyme replacement therapies have primarily targeted internal organs but have been unable to cross the blood–brain barrier, limiting their effectiveness in treating neurological manifestations of the disease. KLOTILIA (verenafusp alfa) is among the first innovative therapies capable of crossing the blood–brain barrier and delivering treatment directly to the brain.
- The therapy received approval from the U.S. Food and Drug Administration (FDA) in 2023. Its introduction in Azerbaijan, made possible through the support of the manufacturer, marks an important step forward in the treatment of metabolic disorders and broadens access to cutting-edge therapies within the country.
- According to Dr. Laman Alirzayeva, the primary goal is to make internationally recognized, evidence-based treatment methods available in Azerbaijan and ensure that children with metabolic disorders can receive world-class medical care without leaving the country.
- This achievement represents not only the beginning of treatment for one child but also a significant milestone in advancing the management of metabolic diseases in Azerbaijan and creating new opportunities for patients and their families.
28 July 2026
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