Announced details
Treatment of Duchenne muscular dystrophy (DMD)
Improvement in TTSTAND velocity (p=0.002); incidence of vertebral fractures of 8.1% versus 41.9% compared with the deflazacort-treated cohort (p=0.0082); 12.17 cm difference in mean height compared with conventional corticosteroids (p<0.0001); comparison of cataract incidence (p=0.015)
Approved and marketed in the United States, European Union, United Kingdom and China
The applicant filing the application for marketing approval in South Korea on behalf of the Company. Nxera Pharma Korea Co., Ltd. is a subsidiary of the Company.
South Korea's pharmaceutical regulatory authority. Other details were not disclosed.
Orphan Drug Designation (ODD) and priority review designation (GIFT)
Ministry of Food and Drug Safety (MFDS) of South Korea
Seoul, the company’s main base
No impact on consolidated full-year results for the fiscal year ending December 2026. Over the medium to long term, the Company believes it will contribute to higher business revenue and corporate value.
The company does not address Contract Value / Offering price.
References including the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2026, the European Medicines Agency and Neurology 2024
| Item | Details |
|---|---|
| Names of Products / Services Covered | vamorolone (treatment for Duchenne muscular dystrophy) |
| Counterparty | Ministry of Food and Drug Safety (MFDS) of South Korea |
| Counterparty overview | South Korea's pharmaceutical regulatory authority. Other details were not disclosed. |
| Relationship with the Company | The applicant filing the application for marketing approval in South Korea on behalf of the Company. Nxera Pharma Korea Co., Ltd. is a subsidiary of the Company. |
| Launch date | During 2028 (scheduled; launch in South Korea) |
| Applicable area | Treatment of Duchenne muscular dystrophy (DMD) |
| Award/certification name | Orphan Drug Designation (ODD) and priority review designation (GIFT) |
| Awarding / certifying body | Ministry of Food and Drug Safety (MFDS) of South Korea |
| Location | Seoul, the company’s main base |
| Quantitative Effects | Improvement in TTSTAND velocity (p=0.002); incidence of vertebral fractures of 8.1% versus 41.9% compared with the deflazacort-treated cohort (p=0.0082); 12.17 cm difference in mean height compared with conventional corticosteroids (p<0.0001); comparison of cataract incidence (p=0.015) |
| Usage history | Approved and marketed in the United States, European Union, United Kingdom and China |
| Impact on Business Performance | No impact on consolidated full-year results for the fiscal year ending December 2026. Over the medium to long term, the Company believes it will contribute to higher business revenue and corporate value. |
| Source | References including the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2026, the European Medicines Agency and Neurology 2024 |
Items this document does not state (5)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.To provide a new treatment option for patients with DMD in South Korea. The Company stated that designation as an orphan drug and under the priority review system is expected to shorten the review period and enable earlier patient access.