CDSCO Approves Hunterase in India to Treat Rare Hunter Syndrome

The Central Drugs Standard Control Organisation has approved Hunterase (idursulfase beta) in India, expanding treatment options for patients diagnosed with Hunter syndrome. Medical specialists in Bengaluru noted that the regulatory approval represents a significant step for Indian patients, given the limited therapies currently available for the rare condition.

Hunter syndrome is a progressive, rare, X-linked recessive genetic disorder that leads to the accumulation of glycosaminoglycans in the body and affects multiple organs. Without ongoing medical care, patients can experience worsening problems affecting growth, bones, joints, breathing, hearing, heart function, and other organs.

According to Dr Vrajendu BB, Consultant in Paediatrics, the rate of progression varies among children. In severe forms, the condition may cause developmental regression and deterioration of cognitive function. Untreated patients with severe cases face a life expectancy of roughly 10 to 20 years, while those with mild forms may live up to 40 years.

The newly approved medication is administered intravenously, typically on a weekly schedule. Medical experts explained that although variants of the drug have previously existed, clinical research and trials have shown improved outcomes for patients using idursulfase beta.

Dr Pramila Kalra, Senior Consultant in the Department of Endocrinology at a private hospital, noted that the disease can cause cardiovascular, neurological, and endocrinological complications. Initiating the treatment in childhood or immediately following diagnosis can help prevent further health complications.

Tests evaluating idursulfase have demonstrated reductions in urinary GAG levels and decreased enlargement of the liver and spleen. Dr Basavaraj S Kumbar, Senior Consultant in Internal Medicine, stated that the realistic medical expectation is the stabilisation or improvement of physical symptoms and a deceleration of disease progression, rather than a complete reversal of the condition. Doctors emphasised that early diagnosis remains critical to preventing irreversible damage.