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deflazacort (Emflaza / MP 104 / MP104)

✓ Approved

Marathon Pharmaceuticals · NR3C1 · 类固醇

什么是 deflazacort？

deflazacort 是一种类固醇，由Marathon Pharmaceuticals研发。该药已获批，用于治疗相关适应症，给药途径：Oral (PO)。

药物档案

商品名Emflaza, MP 104, MP104
公司Marathon Pharmaceuticals
药物类别类固醇, 小分子
分子靶点NR3C1
给药途径Oral (PO)
状态Approved
来源： ClinicalTrials.gov, Marathon Pharmaceuticals

作用机制

分子靶点

deflazacort 作用于 1 个分子靶点：

NR3C1nuclear receptor subfamily 3 group C member 1 (GR, GCCR)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

deflazacort 针对 2 个适应症，涉及 2 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersDuchenne muscular dystrophy✓ Approved
Musculoskeletal and connective tissue disordersJuvenile idiopathic arthritisPreclinical

相关研究文献

PubMedbioRxiv : the preprint server for biology2026-10-25

The annexin A1 mimetic peptide Ac2-26 promotes inflammation-resolution and stimulates skeletal muscle regeneration in the mdx mouse model of Duchenne muscular dystrophy.

Rehman Hamood H, Lu Xinyue X, Nasrallah Savannah M SM, Sharma Binayok B et al.

Duchenne muscular dystrophy (DMD) is an X-linked disorder mainly affecting young boys characterized by repeated cycles of myofiber injury/regeneration and chronic muscle inflammation. Although glucocorticoids can slow disease progression, their potential long-term adverse effects highlight the need for new steroid-sparing therapies for DMD. Annexin A1 (ANXA1) is a glucocorticoid-induced pro-resolving protein that signals primarily through formyl peptide receptor 2 (FPR2). We investigated whether the ANXA1 mimetic peptide Ac2-26 could reduce inflammation, stimulate tissue regeneration, and improve skeletal muscle function in muscular dystrophy. 3-week-old dystrophin-deficient male mdx mice were randomized to receive daily intraperitoneal injections of vehicle control, deflazacort (1 mg/kg/day), or Ac2-26 (1 mg/kg/day) for a duration of 5-weeks. Body composition, skeletal muscle mass, contractile function, and tissue histopathology/immune-cell infiltration were assessed at 8-weeks of age. Direct effects on myogenesis were also examined in vitro using primary D2- mdx myoblasts. ANXA1 was expressed predominantly by myeloid cells rather than resident dystrophic muscle cells. Both deflazacort and Ac2-26 markedly reduced intramuscular inflammation and increased myofiber size. However, Ac2-26 produced broader anabolic and functional benefits, increasing body-weight gain, lean body mass, hindlimb muscle weights, and absolute contractile force. Ac2-26 treatment also directly promoted myogenesis of dystrophic myoblasts in vitro , while pharmacological FPR2 blockade impaired myotube development. Overall, Ac2-26 mimicked the anti-inflammatory effects of deflazacort while providing superior benefits for body weight and composition, skeletal muscle growth, myofiber regeneration, and absolute muscle strength. These findings support therapeutic targeting of the ANXA1-FPR2 axis as a potential safe and effective steroid-sparing immunomodulatory strategy for DMD.

PMID 42926554
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PubMedJournal of medicinal chemistry2026-10-23

Molecular Hybrids of Deflazacort and H2S Donors: From Bench to Potential Treatment for Duchenne Muscular Dystrophy.

Casale Veronica V, Sparaco Rosa R, Cinque Pierfrancesco P, Smimmo Martina M et al.

Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disorder characterized by oxidative stress, chronic inflammation, and skeletal muscle degeneration. Although deflazacort is the standard treatment, its long-term use is associated with significant adverse effects. Since impaired hydrogen sulfide (H2S) signaling contributes to DMD pathogenesis, we designed five novel deflazacort-H2S donor hybrids (I-V) through a molecular hybridization strategy combining anti-inflammatory efficacy with controlled sulfide release. All hybrids showed chemical stability under physiological and acidic conditions, while serum stability varied according to the linker and donor structure. Compound IV exhibited the longest half-life and a slow, l-cysteine-dependent H2S release profile. In LPS + IFN-γ-stimulated C2C12 myotubes, compound IV reduced (i) NOx production, (ii) expression of pro-inflammatory and redox-related genes, and (iii) intracellular ROS levels. In vivo, compound IV, at half of the deflazacort dose, restored locomotor performance, providing superior protection against oxidative stress, highlighting its potential as a dose-sparing therapeutic candidate for DMD.

PMID 42917938
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PubMedFrontiers in neurology2026-09-25

A structured approach to address variability in the global management of adrenal insufficiency in Duchenne muscular dystrophy: the 2025 PJ Nicholoff steroid protocol.

Sbrocchi Anne Marie AM, Ahmet Alexandra A, Kinnett Kathi K, Lautatzis Maria-Elena ME et al.

Adrenal suppression is a potentially life-threatening consequence of chronic glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD), placing individuals at risk for adrenal insufficiency and adrenal crisis during increased physiological stress, missed GC doses, abrupt discontinuation, or transitions between GC regimens. Management is increasingly complex because DMD treatment now includes multiple GC agents, including prednisone/prednisolone, deflazacort, and vamorolone, as well as daily, intermittent, and transition regimens that differ across countries and clinical settings. Although general principles of adrenal insufficiency prevention are well established, implementation remains challenging because of global variability in drug availability, institutional stress-steroid protocols, clinician experience, and caregiver education. This article discusses key sources of variability in adrenal suppression management for individuals with DMD and presents the 2025 PJ Nicholoff Steroid Protocol as an example of a practical, DMD-specific framework. The updated protocol incorporates newer therapies such as vamorolone and emphasizes anticipatory education, written stress-steroid plans (including a weight-based approach), safe GC tapering, confirmation of hypothalamic-pituitary-adrenal axis recovery, and careful management of transitions between GC agents and regimens. This structured approach aims to support safer and more consistent adrenal suppression management for individuals with DMD receiving chronic GC therapy through six critical concepts.

PMID 42787106
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PubMedJournal of neurology, neurosurgery, and psychiatry2026-08-24

Balancing Benefits and Risks: Long-term Use of Daily versus Intermittent Corticosteroids in Duchenne Muscular Dystrophy.

Ikelaar Nadine A NA, Stoop Mees H P MHP, van der Holst Menno M, van Zwet Erik W EW et al.

Duchenne muscular dystrophy (DMD) is treated with intermittent or daily corticosteroid (CS) regimens. Risk-benefit assessment is essential to optimise treatment choice, but long-term efficacy and side effects accumulate beyond clinical trial duration. We describe long-term effects of intermittent compared with daily regimens using real-world data. We compared retrospective data from individuals with DMD treated daily with deflazacort versus intermittently (10 days on/off) with prednisone or deflazacort at three neuromuscular centres in Belgium and the Netherlands between 1995 and 2022. Outcomes included motor, pulmonary, cardiac, anthropometric and bone health domains, analysed using linear mixed effects models and beta regression. Data included 75 patients treated daily and 144 treated intermittently (median age start CS 5.6 years vs 5.5 years, mean follow-up 10.7 years vs 9.5 years). Daily treatment showed later loss of ambulation (15.0 years vs 11.0 years, p<0.001), slower decline of upper and lower limb motor function (p<0.001) and less spinal surgery for scoliosis (16.0% vs 27.1%, p<0.05). Individuals on intermittent treatment were taller (170.2 cm vs 140.8 cm at age 18, p<0.001), had higher bone mineral density Z-scores (p<0.001) and experienced fewer vertebral fractures (p<0.001). Body mass index across all ages, age at forced vital capacity <60% of predicted and annual decline in cardiac function were similar. Daily CS resulted in significantly slower decline in motor function at the expense of reduced height and increased fracture risk. These findings from real-world long-term data reveal that differences between treatment regimens increase over time, highlighting the critical role of such data in guiding individualised shared decision-making.

PMID 42637535
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PubMedMedicine2026-07-18

Disproportionality analysis of sex-stratified adverse event signals in growth impairment: Insights from the FDA adverse event reporting system.

Junyu Xu X, Qian Chen C, Jingnan Xue X, Luying Qi Q et al.

This study aimed to examine sex-disaggregated adverse event signals associated with growth impairment in pediatric patients, utilizing data from the FDA adverse event reporting system, with a particular focus on growth hormone and related therapeutic agents. A disproportionality analysis was performed on FDA adverse event reporting system data spanning 2004 Q1 to 2025 Q1. The analysis encompassed 3281 growth impairment-related reports, disaggregated by gender, employing Reporting Odds Ratios (ROR) and proportional reporting ratios for signal detection, with temporal patterns assessed via Weibull distribution modeling. Significant sex-disaggregated disparities in safety signals were identified. Somatropin exhibited a stronger association with growth impairment in females (ROR 76.85, 95% CI 65.52-90.15) than in males (ROR 37.31, 95% CI 32.99-42.20). Deflazacort showed a male-exclusive signal (ROR 58.25, 95% CI 41.27-82.21), while imatinib displayed a higher risk in females (ROR 15.55, 95% CI 11.29-21.42) compared to males (ROR 4.17, 95% CI 2.91-5.99). Temporal analysis revealed an early-failure pattern, with 50.6% of events occurring within 180 days. These findings generate the hypothesis that sex-disaggregated pharmacovigilance in pediatrics, particularly for growth-modulating therapies, may reveal differential reporting patterns. Should these signals be validated in controlled prospective studies, they could inform the development of customized monitoring frameworks and dosing strategies aimed at potentially mitigating risks in hypothesized high-risk subgroups.

PMID 42470033
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PubMedFrontiers in pharmacology2026-07-11

Post-marketing safety surveillance and signal characterization of the novel dissociative steroid Vamorolone in Duchenne muscular dystrophy: a comparative disproportionality analysis based on FAERS data.

Zeng Yining Y, Wu Hao H, Wang Xueting X, Tan Xiyue X et al.

To comprehensively evaluate the real-world post-marketing safety profile of the novel dissociative steroid Vamorolone and perform a comparative analysis against the traditional glucocorticoid deflazacort. Vamorolone-associated adverse event reports were extracted from the FAERS database (2023Q4-2025Q4) without indication restrictions to maximize detection sensitivity. Disproportionality analysis utilized ROR, PRR, BCPNN, and MGPS, reinforced by a strict Bonferroni correction to assess statistical robustness against multiple testing. Signal novelty was systematically assessed against the official FDA prescribing information. Temporal onset was evaluated via the Weibull distribution. To rigorously compare safety profiles across skeletal, growth, endocrine, and psychiatric domains, an exploratory head-to-head analysis was conducted using 2 × 2 contingency tables to calculate crude odds ratios against deflazacort. We identified 1,171 cases (97.0% explicitly confirmed for muscular dystrophy) comprising 2,860 reported adverse events. Multi-algorithm consensus screening yielded a final refined cohort of 60 potential adverse event signals, predominantly concentrated within psychiatric disorders, investigations, and infections. Label cross-referencing revealed known risks (e.g., stubbornness, EBGM = 163.78 and acute adrenocortical insufficiency, EBGM = 71.14) and potentially novel signals (e.g., enuresis, EBGM = 42.40 and troponin I increased, EBGM = 39.54) that maintained statistical robustness under the stringent Bonferroni threshold. AEs exhibited an "early failure" onset (β = 0.74), particularly rapid for psychiatric events (median 36.5 days, β = 0.57). Comparatively, Vamorolone showed significantly lower reporting odds than deflazacort for growth retardation (Crude OR = 0.15, P = 0.003), osteoporosis (Crude OR = 0.27, P = 0.02), and femur fracture (Crude OR = 0.38, P = 0.008), while psychiatric signals remained comparable (P > 0.05). Exploratory analyses generate the hypothesis that Vamorolone may be associated with lower reporting odds for skeletal and growth-related adverse events compared to deflazacort. Nevertheless, known risks and unexpected hypothesis-generating signals-such as notable HPA axis suppression and early-onset psychiatric disturbances-remain critical safety concerns. Clinicians should interpret these differential reporting patterns with caution and rigorously monitor neurobehavioral and adrenal health during the first three months, with further external validation warranted.

PMID 42434136
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