Seoul National University Hospital (SNUH) has become the first local hospital to administer Biogen’s Spinraza, a treatment for spinal muscular atrophy (SMA), to a pediatric patient after the drug received reimbursement last month.

Biogen’s spinal muscular atrophy treatment Spinraza

Professor Chae Jong-hee announced that she administered the treatment to a two-year-old infant suffering from SMA, who had been losing his exercise function after the diagnosis. Until the launch of the drug in 2016, patients suffering from SMA had only been able to receive spinal interventional treatment, such as relieving discomfort, feeding tube and respiratory support.

The drug has shown excellent clinical performance and survival rate during a clinical trial conducted on 260 patients around the globe, including Korea.

“A drug with excellent efficacy has been developed to deliver hope to patients with rare diseases,” Professor Chae said. “Early diagnosis of SMA, including screening of newborns, is important as the treatment is more effective if started early.”

SMA is a rare neuromuscular disorder, in which muscles gradually contract due to damage in motor neurons in the spinal cord and brain stem. Cognitive function stays normal, but all muscles grow weak, making it difficult to perform basic tasks for life such as breathing or eating. There are about 150 patients in Korea.

However, Spinraza is still one of the most expensive drugs in the world. Even in Korea, where the drug has the cheapest price tag, it has an astronomical price of 92.3 million won ($81,000) a vial.

Although the drug price is close to 100 million won, the financial burden for patients is not as heavy as it receives additional benefit from the nation’s special treatment system for rare and severe incurable diseases. The actual patient copayment is around 810,000 to 5.8 million won a year depending on their income levels.

Experts, however, estimate that the amount of health insurance needed to reimburse the drug will reach tens of billions of won per year as patients have to take four loading doses during their initial treatment and a maintenance dose once every four months after that.

Under the reimbursement plan, patients can now receive insurance benefits if they have the three criteria: diagnosis confirming survival motor neuron 1 gene deletion or mutation, developed SMA-related clinical symptoms and signs at 3 or younger, and are not using a permanent ventilator (more than 16 hours per day or more than 21 days in a row).

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