MFDS Approves Spinal Muscular Atrophy Gene Therapy 'Zolgensmaju' in Korea

[Image source=Yonhap News]

[Asia Economy Reporter Seo So-jeong] The Ministry of Food and Drug Safety (Minister Kim Gang-rip) announced on the 28th that it has approved 'Zolgensma-ju,' a gene therapy for spinal muscular atrophy applied for approval by Korea Novartis, as the second advanced biopharmaceutical under the 'Advanced Regenerative Bio Act' since its enforcement.

Spinal muscular atrophy is a rare disease occurring in approximately 1 in 10,000 newborns worldwide.

Zolgensma-ju is a gene therapy containing genetic material that functionally replaces defective genes and is classified as an advanced biopharmaceutical. It is a treatment administered intravenously in a single dose to patients with spinal muscular atrophy, a degenerative neurological disease caused by mutations or dysfunction of the survival motor neuron 1 (SMN1) gene.

Patients receive the human survival motor neuron (hSMN) gene via a vector to replace the non-functioning SMN1 gene, enabling the production of survival motor neuron (SMN) protein in the central nervous system motor neurons.

This drug is approved for use in patients with spinal muscular atrophy who have biallelic mutations in the SMN1 gene and either a clinical diagnosis of type 1 spinal muscular atrophy or three or fewer copies of the survival motor neuron 2 (SMN2) gene.

As a pharmaceutical product subject to 'long-term follow-up investigation' under Article 30 of the Advanced Regenerative Bio Act, adverse events must be tracked for 15 years from the date of administration, and the contents and results of the long-term follow-up investigation must be reported annually to the Ministry of Food and Drug Safety starting from the first day of sale.

The Ministry of Food and Drug Safety stated, "With this product approval, we expect to provide new treatment opportunities for patients with the fatal disease spinal muscular atrophy."