Nxera Pharma Announces Vamorolone Selected for South Korea’s Accelerated Reimbursement Pilot Program for Rare Disease Treatments

• Vamorolone is one of five treatments selected for the rare disease reimbursement pilot program.
• The program aims to shorten the National Health Insurance listing process to within 100 days.
• Together with GIFT priority review, the accelerated reimbursement pathway could enable a South Korean launch in 2027, earlier than the previously announced target of 2028.

Tokyo, Japan and Cambridge, UK, 6 November 2026 – Nxera Pharma Co., Ltd. (“Nxera” or the “Company”) announces that vamorolone, a treatment for Duchenne muscular dystrophy (DMD), has been selected for the South Korean government’s Accelerated Reimbursement Pilot Program for Rare Disease Treatments. Vamorolone is one of five treatments selected for the pilot program. The program aims to improve access to rare disease treatments by streamlining the National Health Insurance (NHI) listing process following regulatory approval, with a target duration of no more than 100 days. It combines simplified pre-listing assessment and pricing procedures with post-listing reassessment based on real-world evidence (RWE), supporting earlier patient access alongside continued evaluation of therapeutic value.

On 22 October 2026, Nxera announced the submission of a Marketing Authorization Application (MAA) for vamorolone to South Korea’s Ministry of Food and Drug Safety (MFDS). Vamorolone has already received Orphan Drug Designation (ODD) and Global Innovative Products on Fast Track (GIFT) designation in South Korea and is currently under regulatory review. Taking into account priority review under GIFT, Nxera is targeting regulatory approval in the third quarter of 2027. The accelerated reimbursement pathway, together with GIFT priority review, could enable Nxera to bring forward the commercial launch of vamorolone in South Korea to 2027 from the previously announced target of 2028.

The Company will work closely with the relevant South Korean authorities to advance regulatory approval, reimbursement and product supply preparations, with the aim of launching vamorolone concurrently with its NHI listing. The timing of launch will depend on the progress of regulatory review and reimbursement procedures. Following NHI listing and commercial launch, real-world data (RWD) from Korean patients is expected to be collected through a patient registry established by the Health Insurance Review & Assessment Service (HIRA). The resulting RWE is expected to support reassessment of vamorolone’s long-term clinical outcomes and therapeutic value.

Mr. MinBok Lee, President and Representative Director of Nxera Pharma Korea, commented: " Selection for this pilot program is an important step towards giving patients with DMD and their families in South Korea earlier access to a new treatment option. Together with the GIFT priority review pathway, it could help us shorten the time from regulatory review to reimbursed availability of vamorolone. We are grateful to Santhera Pharmaceuticals for the scientific evidence and close collaboration that have supported this progress. We will continue to work with the Korean authorities and our partners to secure regulatory approval, complete reimbursement procedures and establish a reliable supply of vamorolone for patients.”

About South Korea’s Accelerated Reimbursement Pilot Program for Rare Disease Treatments
Introduced by the South Korean government in 2026, the pilot program is designed to improve timely access to medicines for patients with serious rare diseases and to evaluate the feasibility and effectiveness of a new accelerated reimbursement system. Predefined contractual terms replace separate negotiations on drug prices and total pharmaceutical expenditure under the conventional listing process. Prices are set with reference to 90% of the lowest adjusted price across eight reference countries (A8). Reimbursement prices and coverage conditions may subsequently be revised following reassessment of real-world clinical outcomes. The A8 reference countries are the United States, United Kingdom, Germany, France, Italy, Switzerland, Japan and Canada.

About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD typically reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure.

About vamorolone
Vamorolone is a dissociative corticosteroid approved for the treatment of Duchenne muscular dystrophy (DMD). It binds selectively to the glucocorticoid receptor and triggers anti-inflammatory activity through inhibition of NF‑κB-mediated gene transcription, while inducing reduced transactivation of other genes [1]. Vamorolone is not a substrate for 11‑β-hydroxysteroid dehydrogenase (11β-HSD) enzymes, which are involved in the local amplification of glucocorticoid activity in tissues and have been implicated in corticosteroid-associated toxicity [2,3]. This pharmacological profile is the basis for its classification as a dissociative corticosteroid, designed to preserve anti-inflammatory efficacy while reducing the systemic effects associated with long-term conventional corticosteroid therapy [1–3].

In the pivotal Phase 2b VISION-DMD study, vamorolone met its primary endpoint, demonstrating a statistically significant improvement in Time to Stand (TTSTAND) velocity versus placebo at 24 weeks (p = 0.002) [4]. The most commonly reported adverse reactions were cushingoid features, vomiting, weight increase, increased appetite, and irritability; most were mild to moderate in severity [1].

Long-term data from up to eight years of vamorolone treatment were presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March 2026[5,6]. In propensity-matched analyses, vamorolone demonstrated durable efficacy comparable to standard-of-care corticosteroids and a differentiated safety profile: a lower incidence of vertebral fractures versus deflazacort-treated cohorts (8.1% vs 41.9%; p = 0.0082) [5]; maintained normal growth trajectory with a mean height advantage of 12.17 cm versus conventional corticosteroids (p < 0.0001) [5,6], and a lower incidence of cataracts versus deflazacort (p = 0.015), with no observed cases of glaucoma [5].

References

[1] AGAMREE® (vamorolone) Summary of Product Characteristics. European Medicines Agency; authorised 14 December 2023.
[2] Heier CR, Damsker JM, Yu Q, et al. VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. Life Sci Alliance. 2019;2(1): e201800186.
[3] Reeves EKM, Hoffman EP, Nagaraju K, et al. VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem. 2013; 21(8): 2241–2249.

[4] Dang UJ, Damsker JM, Guglieri M, et al. Efficacy and safety of vamorolone over 48 weeks in boys with Duchenne muscular dystrophy (VISION-DMD). Neurology. 2024;102(5): e208112.
[5] Guglieri M, et al. Long-term impact of vamorolone on bone health compared to standard of care glucocorticoids in boys with Duchenne muscular dystrophy. Poster 62S, MDA Clinical & Scientific Conference 2026.
[6] McDonald CM, et al. Comparative analysis of long-term effectiveness of vamorolone versus standard of care glucocorticoid treatment in boys with Duchenne muscular dystrophy. Poster 23S, MDA Clinical & Scientific Conference 2026.

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About Nxera Pharma
Nxera Pharma is a technology powered biopharma company in pursuit of new specialty medicines to improve the lives of patients with unmet needs in Japan and globally. The Company has built an agile, new-generation commercial business in Japan to develop and commercialize innovative medicines, including several launched products, to address this high-value, large and growing market and those in the broader APAC region. In addition, the Company is advancing an extensive pipeline internally and in partnership with leading pharma and biotech companies powered by its unique NxWave™ GPCR structure-based drug discovery platform. Nxera Pharma operates at key locations in Tokyo and Osaka (Japan), London and Cambridge (UK), Basel (Switzerland) and Seoul (South Korea) and is listed on the Tokyo Stock Exchange (ticker: 4565).

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