Emflaza (deflazacort) / Marathon, PTC Therap - LARVOL DELTA

Eliminating Saliva Contamination in Exhaled Breath Condensate (EBC) (ERS 2025) - " With an additive-sealed saliva trap, EBC yields (µL/min) were consistent across interfaces (15min - MP:124.7±11.7, FM:113.7±15.3; 30min - MP:104.7±5.7, FM:103.1±7.2), but facemasks reduced (p<0.01) fluid buildup in the trap (15min:0µL; 30min:3.3±5.8µL) vs. mouthpiece use (15min:333.3±145.7µL, 30min:505.0±366.3µL)... Using facemasks minimised saliva accumulation in the saliva trap, and fully-sealed traps prevented wicking, to eliminate salivary contamination in EBC. These methods enable uncontaminated EBC collection without affecting sampling rates, potentially improving biomarker sensitivity and diagnostic outcomes. 1 Horváth I, Barnes PJ, Loukides S, et al."

Pulmonary Disease • Respiratory Diseases

Why It Is Still Important to Ensure the Price is Right: Updates to Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy (ISPOR 2025) - "Corticosteroids (Emflaza and Amagree) are excluded as they have significantly different value and price considerations. Coverage criteria aligned to trial criteria is to ensure appropriate use. Duvyzat’s parity access to existing agents is likely a product of similar price-value alignment. If clinical benefit is unclear and price is high (e.g., non-ambulatory Elevidys), there is a prevailing risk of stakeholders adding restrictions (e.g., step edits) or only approving on a case-by-case basis (i.e., non-formulary)."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading. (PubMed, Orphanet J Rare Dis) - "Several sources of cardiac disease heterogeneity are well-studied in patients with DMD. Yet, the certainty of evidence is generally low, and little is known of the contribution of non-pharmacological interventions, as well as the impact of different criteria for initiation of specific treatments. Our findings help raise awareness of prevailing unmet needs, shape expectations of treatment outcomes, and inform the design of future research."

Journal • Review • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Duchenne Muscular Dystrophy • Genetic Disorders • Heart Failure • Muscular Dystrophy • Rare Diseases

Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza Oral Suspension (deflazacort) for Duchenne Muscular Dystrophy (Businesswire) - "Cranbury Pharmaceuticals...announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza oral suspension (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD). The generic deflazacort oral suspension is now available to patients in the United States."

FDA approval • Duchenne Muscular Dystrophy • Genetic Disorders

Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis. (PubMed, J Neuromuscul Dis) - "Glucocorticoid therapy was associated with delayed loss of ambulation (overall meta-analysis HR deflazacort/prednisone/prednisolone: 0.44 [95% CI: 0.40-0.48]) (n = 25 studies)...Treatment with ataluren (n = 2 studies) and eteplirsen (n = 3 studies) were associated with prolonged ambulation...In total, 33% of studies exhibited some risk of bias. Our synthesis of predictors of loss of ambulation in DMD contributes to the understanding the natural history of disease and informs the design of new trials of novel therapies targeting this heavily burdened patient population."

IO biomarker • Journal • Retrospective data • Review • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CD40 • SPP1

Utilization, Expenditure and Price of Muscular Dystrophy-Duchenne Medications in the US Medicaid Programs: Trends From 2017 to 2022 (ISPOR 2024) - "Five study medications were Exondys, Emflaza, Vyondys, Viltepso, and Amondys. As DMD medications increasingly manage this severe disease, the spending and utilization have also increased greatly. The study findings help in providing insightful details about the cost drivers of DMD medications to be explored in health policies."

Medicaid • Reimbursement • US reimbursement • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Why It Is Important to Ensure the Price Is Right: Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy (ISPOR 2024) - "While most of the top plans by lives covered allow the use of most DMD therapies, some smaller plans do not cover therapies such as Elevidys or Viltepso. ICER analysis found two DMD therapies (Emflaza and Exondys 51) providing low long-term value but still recommend payers to cover the therapies with prior authorizations... As the DMD disease landscape continues to evolve, manufacturers must ensure price is supported by value and data, regardless of being indicated in a rare space. This importance only intensifies as more therapies become available. While most plans currently cover DMD therapies, if value and price are perceived to be misaligned, stakeholders can attempt to restrict access to who they determine as the most appropriate patients for treatment (i.e., population studied in the trial) or only approve on a case-by-case basis (i.e., non-formulary)."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Characterization of deflazacort use in young Duchenne muscular dystrophy patients: an analysis of data from the PTC Cares database (MDA 2024) - "These data provide insights into the characteristics of young patients receiving deflazacort in the US and identify discrepancies in referral rates of these patients between regions. Further analyses are ongoing and updated data will be presented in the poster."

Clinical • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis (MDA 2024) - P, P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=277) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use... These real-world, multi-country data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Age at loss of ambulation in patients with DMD from the STRIDE Registry and the CINRG Natural History Study: a matched cohort analysis (MDA 2024) - P, P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=277) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use... The study showed that in routine clinical practice ataluren plus SoC significantly delayed age at LOA compared with SoC alone in patients with nmDMD."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Comparison of pharmaceutical properties and biological activity of prednisolone, deflazacort, and vamorolone in DMD disease models. (PubMed, Hum Mol Genet) - "The corticosteroids prednisone/prednisolone and deflazacort are used to treat DMD as the standard of care; however, only deflazacort is FDA approved for DMD...The results of these studies indicate that efficacious doses of vamorolone, are associated with similar side effects as seen with other corticosteroids. Further, because vamorolone is not a strong P-gp substrate, vamorolone distributes into the CNS increasing the potential CNS side-effects."

Journal • CNS Disorders • Depression • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Psychiatry • BGLAP

Delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Age at loss of ambulation in patients with DMD from the STRIDE Registry and the CINRG Natural History Study: a matched cohort analysis (EPNS 2023) - P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use... These Kaplan-Meier analyses showed that in routine clinical practice ataluren plus SoC delayed age at LOA by 5.4 years compared with SoC alone in patients with nmDMD."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis (EPNS 2023) - P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use... These interim registry data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Evaluation of the Biodistribution, Efficacy, and Side-Effect Profile of Deflazacort, Prednisolone and Vamorolone in a DMD Mouse Model (AAN 2023) - "Deflazacort was the most effective in increasing muscle strength, as measured by the grip test. Furthermore, of the three corticosteroids, deflazacort resulted in the fewest behavioral side-effects."

Adverse events • Preclinical • CNS Disorders • Depression • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Psychiatry

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status. (PubMed, J Comp Eff Res) - " Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1. (PubMed, J Neuromuscul Dis) - "Steroid use after loss of ambulation in DMD was associated with delayed progression of important pulmonary, cardiac, and upper extremity functional deficits, suggesting some benefits of deflazacort over prednisone."

Clinical data • Journal • Cough • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Respiratory Diseases

Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: A matched cohort analysis (WMS 2022) - P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use...Kaplan–Meier analyses showed that ataluren plus SoC delayed age at LOA compared with SoC alone (p<0.0001). These Kaplan–Meier analyses showed that in routine clinical practice ataluren plus SoC delayed age at LOA by 5.4 years compared with SoC alone in patients with nmDMD."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis (WMS 2022) - P=N/A | "Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use...Median (95% CI) ages at %-predicted FVC <30% (STRIDE vs CINRG) were not estimable and 22.5 (20.3, 25.4) years, respectively (p=0.0008). These interim registry data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) (clinicaltrials.gov) - P3 | N=11 | Terminated | Sponsor: PTC Therapeutics | N=30 ➔ 11 | Completed ➔ Terminated; The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.

Enrollment change • Trial termination • Muscular Dystrophy

Associations Between Steroid Treatment and Clinical Outcomes Among Non-ambulatory Patients with Duchenne Muscular Dystrophy (DMD) (AAN 2022) - "Associations between steroid treatment (prednisone, deflazacort, or no steroids) and pulmonary, cardiac, and functional outcomes were assessed, including changes in forced vital capacity [FVC] %-predicted, left ventricular ejection fraction [LVEF], performance of upper limb [PUL] score, and loss of hand-to-mouth function... Steroid use after loss of ambulation was associated with delayed progression of important pulmonary, cardiac and functional deficits in DMD."

Clinical • Clinical data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Associations Between Deflazacort Versus Prednisone/Prednisolone and Markers of Disease Progression in Clinically Important Subgroups of Patients with Duchenne Muscular Dystrophy (AAN 2022) - P2b, P3 | "Benefits of daily deflazacort compared to daily prednisone for preserving ambulatory function in DMD were most evident among patients who were older, had been on steroids longer, or were at a more progressed disease stage. These results add to the evidence for a potential cumulative benefit of deflazacort versus prednisone."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. (PubMed, J Comp Eff Res) - P2b, P3 | "Materials & Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests)...Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy."

Clinical • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment. (PubMed, Muscle Nerve) - P3 | "Unlike fixed-time analyses, this analytical approach enabled demonstration of cumulative, longitudinal treatment effects over time using repeatedly measured NSAA observations."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Ring C-chair and boat conformation of morphinan template dictates functional selectivity at opioid receptors (ACS-Fall 2021) - "Morphine and other clinically used opioid agonists that target the mu-opioid receptor (MOR) remain the preferred treatment of moderate to severe pain...We had previously developed a morphinan based ligand named MP1104 which showed picomolar affinity at kappa opioid receptor (ACS Chem Neuro 2015, PMID: 26325040)...The lead compounds, MP1207 and MP1208, displayed MOR/KOR Gi-partial agonism with diminished arrestin signaling, showed efficient analgesia with attenuated liabilities, including respiratory depression and conditioned place preference and aversion in mice. The findings validate a novel structure-inspired paradigm for achieving beneficial in vivo profiles for analgesia through different mechanisms that include, partial agonism, and dual MOR/KOR agonism."

Addiction (Opioid and Alcohol) • CNS Disorders • Depression • Pain • Psychiatry