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Korea Approves Voxzogo for Pediatric Chondrodysplasia Treatment

The Korean Ministry of Food and Drug Safety (MFDS) has approved Voxzogo (vosoritide) for treating chondrodysplasia in children aged four months and older. This breakthrough therapy, developed by BioMarin Pharmaceutical, represents the first treatment option for pediatric chondrodysplasia patients in Korea, offering new hope for those affected by this rare genetic disorder.

The Korean Ministry of Food and Drug Safety (MFDS) has granted approval for Voxzogo Injection (vosoritide), marking a significant milestone as Korea's first therapeutic option for pediatric chondrodysplasia. The treatment is indicated for children aged four months or older whose growth plates remain open.

Mechanism of Action and Disease Background

Chondrodysplasia, a genetic disorder affecting bone growth, stems from mutations in the FGFR-3 (fibroblast growth factor receptor-3) gene. This crucial gene plays a vital role in regulating chondrocyte proliferation and differentiation. Voxzogo's mechanism of action centers on inhibiting FGFR-3 signaling, thereby promoting chondrocyte proliferation and differentiation, which ultimately enhances bone formation within cartilage tissue.

Global Regulatory Progress

The drug's approval in Korea follows significant regulatory achievements in other markets. BioMarin Pharmaceutical, the U.S. biotech company behind Voxzogo, initially secured FDA accelerated approval in November 2021 for patients aged five years and older. In a notable expansion of its therapeutic reach, the FDA broadened the indication in October 2023 to include children under five, effectively making the treatment available to pediatric patients across all age groups.

Strategic Fast-Track Designation

Highlighting its innovative potential, the MFDS designated Voxzogo as the 10th product under the Global Innovative Product Fast Tracking Support System (GIFT) in July 2023. The drug has been introduced to the Korean market through Samoh Pharm, serving as the local distribution partner.

Impact on Patient Care

"Previously, there was no treatment for pediatric chondrodysplasia patients, but with the approval of this treatment, it is expected to provide new treatment opportunities for pediatric chondrodysplasia patients," stated ministry officials. The MFDS has emphasized its commitment to expediting access to novel treatments for rare and challenging diseases, leveraging their regulatory science expertise to expand therapeutic options for patients in need.
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Reference News

[1]
Korea approves rare pediatric chondrodysplasia drug Voxzogo < Policy < Article - KBR
koreabiomed.com · Jan 2, 2025

Korea approved Voxzogo Injection, the first pediatric chondrodysplasia drug, for children aged four months or older with...

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