Novartis Korea said Monday that it released Zolgensma, the nation's first gene replacement treatment for spinal muscular atrophy (SMA), on Monday with the registration of health insurance benefits.
SMA is a fatal rare genetic disease and one of the main causes of infant death, in which muscles gradually contract due to lack or mutation of the normal SMN1 gene.
Patients newly diagnosed with SMA with double allele mutations in the SMN1 gene, genetic diagnosis of defects, or mutations in the 5q SMN1 gene are eligible for Zolgensma’s insurance coverages. Additionally, patients with a clinical diagnosis of SMA type 1 or with less than two clones of the SMN2 gene or less than 9-12 months old at the time of administration can also receive these benefits.
This year, patients administering existing SMA therapeutics will also temporarily be eligible for reimbursement if they are replaced with Zolgensma.
“STR1VE" and "SPR1NT" clinical trials confirmed an unprecedented survival level and rapid improvement in motor ability with Zolgensma. Additionally, a long-term observational study of phase 3 clinical trials of “START” conducted on SMA type 1 showed that the therapeutic effect was maintained for more than seven years after administration.
Zolgensma is the first gene replacement treatment for SMA, which can prevent the disease’s progression with one-time intravenous administration. Additionally, the drug’s innovative mechanism whereby SMN proteins are continuously produced and distributed in the body after administration reduces the treatment burden for patients and guardians, and likely supported the insurance coverage decision, said the company.
"I am very pleased with Zolgensma’s insurance benefits, which can prevent the progression of SMA with just one dose,” Professor Chae Jong-hee of Pediatric Neurology at Seoul National University Hospital, said. “Since Zolgensma has already shown amazing treatment effects globally, we expect the insurance coverage to be an opportunity to change the treatment paradigm for rare diseases in SMA and Korea.”
Cho Yeon-jin, executive director of Novartis Korea’s ophthalmology division, said, "We are truly happy to deliver good news to SMA patients and their families eagerly waiting for Zolgensma to apply for insurance benefits.”
The drug’s upper limit of health insurance benefits is 2 billion won ($1.53 million) per kit. However, patients have only to pay a maximum of 5.98 million won.