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deflazacort (Emflaza / MP 104 / MP104)

✓ Approved

Marathon Pharmaceuticals · NR3C1 · Steroids

What is deflazacort?

deflazacort is a steroids developed by Marathon Pharmaceuticals. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesEmflaza, MP 104, MP104
CompanyMarathon Pharmaceuticals
Drug ClassSteroids, Small Molecule
Molecular TargetNR3C1
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Marathon Pharmaceuticals

Mechanism of Action

Molecular Targets

deflazacort acts on 1 molecular target:

NR3C1nuclear receptor subfamily 3 group C member 1 (GR, GCCR)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

deflazacort is developed for 2 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersDuchenne muscular dystrophy✓ Approved
Musculoskeletal and connective tissue disordersJuvenile idiopathic arthritisPreclinical

Related Research Articles

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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PubMedCureus2026-07-09

Prescription Patterns in Rheumatoid Arthritis Patients at a Tertiary Care Hospital: A Prospective Cross-Sectional Study.

Mani Kaushalendra K, Jadhav Sujata S, Thorat Vandana V, Gargate Prathmesh P et al.

Rheumatoid arthritis (RA) is a chronic, debilitating, systemic inflammatory condition seen worldwide. Prescribing medications is one of the most common interventions in RA treatment. Therefore, we aimed to study the prescription pattern for RA patients at our tertiary care center.  Methods: This was a prospective, cross-sectional, descriptive study of 95 RA patients. Data on demographic profile and drug prescriptions were collected from the prescriptions given by consultants and then analyzed.  Results: Most patients were aged 41-60 years (66.3%), and females predominated (61.1%). Common comorbidities included hypertension (21.1%), diabetes (16.8%), and anemia (12.6%). Disease duration was mainly three to five years (55.8%). Nearly half had an education below graduation (49.5%); common occupations were housewives (37.9%) and professionals (32.6%). Disease‑modifying anti‑rheumatic drugs (DMARDs) were the most prescribed class (90.5%), followed by nonsteroidal anti‑inflammatory drugs (NSAIDs; 64.2%) and corticosteroids (51.6%); biologics were limited (8.4%). Three‑drug prescriptions were most common (35.8%). Methotrexate was the predominant DMARD (75.8%), while diclofenac (20.0%) and deflazacort (18.9%) were the most frequently used NSAID and corticosteroid, respectively.  Conclusion: RA predominantly affects middle‑aged females with moderate disease duration and a notable comorbidity burden. Treatment patterns show strong reliance on conventional DMARDs, particularly methotrexate, with adjunct use of NSAIDs and corticosteroids, while biologics remain underused.

PMID 42422649
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PubMedJournal of neuromuscular diseases2026-07-04

Disease progression and economic burden of duchenne muscular dystrophy: A retrospective study using Swedish register data.

Sejersen Thomas T, Kroksmark Anna-Karin AK, Törnblom Aina A, Toghanian Samira S et al.

BackgroundDuchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disorder causing progressive muscle loss and early death. Despite global evidence on its burden, data from Sweden remain limited.ObjectiveThis study described the natural history of DMD, treatment patterns, healthcare resource utilization, and direct medical and non-medical costs among patients with DMD and their caregivers in Sweden.MethodsA retrospective, observational matched cohort study was conducted using data from the Swedish National Registry for Neuromuscular Disorders linked to national health and social registers. Patients diagnosed with DMD and their caregivers were compared to matched population controls. Disease progression was classified into four stages based on the HERCULES model.ResultsA total of 211 DMD patients (mean age at diagnosis: 4.9 years) and 877 comparators were included. Patients experienced progressive loss of function, spending on average 3.8, 4.8, 4.8, and 7.4 years in stages 1-4, respectively. Glucocorticoid use was high (84% on either prednisolone or deflazacort). Annual direct medical costs were significantly higher for DMD patients (€14,590 vs. €660). Direct non-medical costs, predominantly for personal assistance, increased substantially with disease stage, representing 89% of total DMD-related costs (€135,671 annually).ConclusionsDMD is a severe disorder marked by prolonged time in the later stages of the disease and increasing need for support. DMD imposes a significant economic burden in Sweden, with direct non-medical costs far exceeding direct medical costs. These findings highlight the need for and value of early and effective interventions to delay disease progression and alleviate societal burden.

PMID 42400353
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PubMedBMC pharmacology & toxicology2026-07-01

Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy.

Ompad Gerard G, Saura Petra Maria PM, Heckmann Nicole Sonne NS, Rossi Andrea A et al.

Currently, there is a lack of meta-analysis providing precise incidence estimates of adverse drug reactions (ADRs) and associated risk differences (RDs) linked to pharmacological treatments for muscle degeneration in Duchenne Muscular Dystrophy (DMD). A systematic search of PubMed, Embase, and ClinicalTrials.gov (January 1, 2000-June 5, 2026) identified clinical trials evaluating safety of pharmacological treatments for muscle degeneration in DMD. A random-effects meta-analysis estimated ADR incidence, and RDs versus a common reference (givinostat) were derived from the pooled incidences rather than from conventional head-to-head treatment contrasts. Thirteen clinical trials were included. Heterogeneity in ADR incidence across treatments (p < 0.05) was identified. Prednisone and prednisolone showed the highest proportions of serious ADRs. Discrepancies were found compared to Summary of Product Characteristics (SmPC): deflazacort showed higher rates of erythema (+ 1.5%) and pollakiuria (+ 4.8%), while vamorolone showed increased Cushingoid features and appetite (+ 7.0%). Subgroup analyses showed dose- and duration-dependent ADRs for deflazacort and prednisone. A reference-anchored exploratory comparison of pooled incidences showed lower gastrointestinal and hematological ADR risks with corticosteroid monotherapy than with givinostat plus corticosteroids. Deflazacort showed lower risks of diarrhea (RD: - 0.3860), decreased appetite (RD: - 0.3160), and decreased platelet count (RD: - 0.4110). Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks.

PMID 42380960
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