Nxera’s Vamorolone Granted Key Regulatory Designations Supporting Faster Access for Duchenne Muscular Dystrophy Patients in South Korea

• Orphan Drug Designation (ODD) recognizes Duchenne muscular dystrophy as a rare disease with significant unmet medical need without an established treatment in South Korea.
• Global Innovative Products on Fast Track (GIFT) designation, a priority review pathway introduced by Korea's Ministry of Food and Drug Safety, may support accelerated regulatory review and faster patient access to innovative therapies in South Korea.
• Nxera plans to submit a Marketing Authorization Application for vamorolone in South Korea during 2026.

Tokyo, Japan and Cambridge, UK, 2 June 2026 – Nxera Pharma Co., Ltd. (“Nxera” or the “Company”)　 announces that the Ministry of Food and Drug Safety (MFDS) of the Republic of Korea has granted Orphan Drug Designation (ODD) and Global Innovative Products on Fast Track (GIFT) designation to vamorolone for the treatment of Duchenne muscular dystrophy (DMD). The Company plans to submit a Marketing Authorization Application (MAA) for vamorolone in South Korea during 2026.

MFDS's ODD framework applies to medicines intended for diseases affecting fewer than 20,000 patients in South Korea, for diseases with no available alternative treatment, or for medicines that demonstrate significantly improved safety and efficacy compared with existing treatments. The ODD for vamorolone was granted following MFDS's determination that the product meets the designation criteria stipulated under the “Regulation on Orphan Drug Designation.” The designation formally recognizes that, in Korea, DMD is a rare disease for which no established treatment is currently available.

The GIFT designation is a priority review pathway introduced by MFDS in September 2022 to accelerate the regulatory review of innovative medicinal products targeting life-threatening or serious diseases, including rare diseases. Under the GIFT framework, the standard MFDS review timeline of 120 working days may be shortened to up to 90 working days through rolling review and close communication with reviewers, potentially helping accelerate patients access to innovative therapies in South Korea. Nxera believes that the GIFT designation for vamorolone will help accelerate access to this medicine for DMD patients in South Korea.

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