Novartis Korea, SMA gene replacement therapy ‘Zolgensma’ Symposium

Symposium view [사진=한국노바티스]
Novartis Korea held a launching symposium for pediatricians and pediatric neurologists on the 24th to commemorate the domestic reimbursement of Zolgensma (Onasemnogeneaveparvovec), the first gene replacement treatment for spinal muscular atrophy (SMA) in Korea. said to have been held.

Zolgensma is the first and only gene replacement treatment in Korea that can prevent the progression of SMA with once-in-a-lifetime intravenous administration. It was developed as a mechanism to solve the root cause of the disease by providing a functional replacement of the SMN1 gene, the cause of SMA. Zolgensma insurance benefits were applied to patients newly diagnosed with SMA from the 1st.

Professor Jonghee Chae of the Department of Pediatrics at Seoul National University Hospital, who chaired the symposium, gave a keynote lecture on ‘New era of Gene Replacement Therapy’.
Professor Chae said, “With the advent of gene replacement therapy, the domestic rare disease treatment environment has reached a new turning point. In the future, gene therapy will lead the medical industry in the field of rare diseases.”

Professor Joanna Jo of the Department of Pediatrics at Seoul National University Bundang Hospital presented ‘How to distinguish SMA from neuromuscular disease’. Professor Cho said that it is very important to suspect a neuromuscular disease in a general pediatric department and quickly conduct a test to diagnose it.

“This Zolgensma launching symposium was a meaningful event to confirm the value of Zolgensma, which will take a new focus in the domestic gene therapy market, and to share the importance of diagnosis and treatment of SMA,” said Yoo Byung-jae, CEO of Novartis Korea.

SMA is a rare and fatal genetic disease in which muscles gradually atrophy due to a deficiency or mutation of the normal SMN1 gene, and is one of the leading causes of infant mortality. It occurs in about 1 in 10,000 newborns worldwide. Type 1, accounting for about 60% of SMA patients, is the most severe type. If left untreated, 90% of patients can die before the age of 2 years.

Zolgensma was approved by the US FDA in 2019 and is currently approved in more than 40 countries. After receiving product approval from the Ministry of Food and Drug Safety in May last year, it entered the right to benefit for the first time among gene replacement therapies.

Reporter Jang Bom-i [email protected]

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