Announced details
Treatment of Duchenne muscular dystrophy (DMD)
GIFT may shorten the standard review period from 120 business days to as short as 90 business days.
The relationship involves regulatory submission and review. The company has a South Korean subsidiary, Nxera Pharma Korea Co., Ltd.
South Korea’s pharmaceutical regulatory authority. It introduced the GIFT priority review system in September 2022.
Orphan drug designation (ODD) and designation under the priority review system (GIFT)
Ministry of Food and Drug Safety (MFDS) of South Korea
Seoul, the company’s main base
The impact on full-year consolidated results for the fiscal year ending December 2026 is expected to be minor. Over the medium to long term, the company believes this will contribute to higher business revenue and corporate value.
The company does not address Contract Value / Offering price.
Ministry of Food and Drug Safety (MFDS) of South Korea, Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2026, and references [1] to [6]
| Item | Details |
|---|---|
| Names of Products / Services Covered | vamorolone |
| Counterparty | Ministry of Food and Drug Safety (MFDS) of South Korea |
| Counterparty overview | South Korea’s pharmaceutical regulatory authority. It introduced the GIFT priority review system in September 2022. |
| Relationship with the Company | The relationship involves regulatory submission and review. The company has a South Korean subsidiary, Nxera Pharma Korea Co., Ltd. |
| Launch date | The company plans to file a marketing authorization application in South Korea during 2026. |
| Applicable area | Treatment of Duchenne muscular dystrophy (DMD) |
| Award/certification name | Orphan drug designation (ODD) and designation under the priority review system (GIFT) |
| Awarding / certifying body | Ministry of Food and Drug Safety (MFDS) of South Korea |
| Location | Seoul, the company’s main base |
| Quantitative Effects | GIFT may shorten the standard review period from 120 business days to as short as 90 business days. |
| Impact on Business Performance | The impact on full-year consolidated results for the fiscal year ending December 2026 is expected to be minor. Over the medium to long term, the company believes this will contribute to higher business revenue and corporate value. |
| Source | Ministry of Food and Drug Safety (MFDS) of South Korea, Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2026, and references [1] to [6] |
Items this document does not state (6)
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.South Korean patients with DMD face a significant unmet medical need, and the designations are intended to accelerate patient access to innovative treatments. ODD indicates that DMD has been recognized in South Korea as a rare disease for which no established treatment exists.