Section 3 of 6
Product information and disease description
74 evidence topics · 59 sources
Product description
Phase of product development
Regulatory submission: Class 2 BLA resubmission submitted February 2026
FDA Advisory Committee
Anticipated FDA action date
Launch
No evidence found.
Special FDA designations
Product information
Generic, brand name and therapeutic class of product
Unapproved product name and drug class
Dosage forms and strengths
Investigational cell suspension
Deramiocel-specific material safety data sheet
No evidence found.
Average sales price and wholesale acquisition cost
Product pricing information: allegation in Capricor complaint concerning its distributor
Marketed-product average sales price and announced wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
No evidence found.
Indication
Refined proposed indication
Pharmacology
Mechanism of action
Pharmacodynamics
Pharmacokinetics
FDA assessment of tissue-distribution uncertainty
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
HOPE-3: studied age and ambulatory population
Renal impairment, hepatic impairment, pregnancy, and lactation
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on Deramiocel
No evidence found.
Effects of Deramiocel on other drugs
No evidence found.
Dosing and administration
Dosage
Administration
Administration setting
Access and distribution
Distribution agreement
Co-prescribed/Concomitant therapies
HOPE-3: background corticosteroid therapy
Effect of Deramiocel on quality measures
Deramiocel-specific effects on quality measures
No evidence found.
Disease-state evidence: implementation of care considerations
Product comparison
HOPE-3: placebo-controlled comparison on background corticosteroids
Head-to-head comparisons with active DMD therapies
No evidence found.
Place of product in therapy
Disease description
Definition and etiology
Epidemiology
Incidence of Duchenne muscular dystrophy
Australian birth cohort: cumulative incidence
Prevalence of Duchenne muscular dystrophy
Global birth prevalence: pooled estimate
Natural history, survival, and mortality
Pathophysiology
Dystrophin deficiency and progressive degeneration
Diagnosis
Genetic testing before mutation-specific treatment
Clinical presentation - signs and symptoms
Motor symptoms: childhood-onset progressive muscle weakness
Cardiac manifestations: cardiomyopathy
Long-term morbidity
Burden of Duchenne muscular dystrophy
Humanistic burden and health-related quality of life
Humanistic burden: Chilean cross-sectional study
Health-state utilities: upper-limb function in non-ambulatory DMD
Economic burden and healthcare resource utilization
Health-state utilities, costs, and healthcare resource use in DMD
United States medical costs: Medicaid and commercial insurance
United States healthcare costs: DMD versus matched non-MD controls
Economic impact of Duchenne muscular dystrophy on families
Caregiver productivity: Europe, Japan, and the United States
Economic impact of diagnostic testing
Approaches to treatment
Current treatment options and standard of care
Corticosteroids
Current corticosteroid options
Gene replacement therapy
Delandistrogene moxeparvovec: revised eligible population
Exon-skipping antisense oligonucleotides
Mutation-specific exon-skipping options
| Drug | Required exon-skipping amenability |
|---|---|
| Casimersen | “skipping exon 45.” |
| Viltolarsen | “skipping exon 53.” |
| Golodirsen | “skipping exon 53.” |
| Eteplirsen | “skipping exon 51.” |
Histone deacetylase inhibitors
Givinostat: therapeutic class and treated age range
Physical and occupational therapy
Rehabilitation: prevention of contracture and deformity
Cardioprotective therapies
Respiratory support
Assisted lung inflation, cough, and ventilation
Bone health therapies
Fracture-related bone protection: treatment options
Nutritional support
Psychosocial support
Limitations of current therapies
Summary
Current treatment combines disease-directed therapy with rehabilitation and organ-specific supportive care. Corticosteroid adverse effects include weight gain, behavioural changes, growth suppression, bone loss, and cataracts. Exon-skipping options require mutations amenable to skipping a specified exon. The revised delandistrogene moxeparvovec indication is limited to ambulatory patients aged four years or older with a confirmed DMD mutation, and FDA identifies risks of serious liver injury and acute liver failure, including fatal outcomes. These eligibility restrictions and adverse effects limit the applicability of available therapies across the DMD population.
Place in treatment, anticipated use, and care setting
Summary
Deramiocel has been studied as an intravenous treatment administered every three months in outpatient settings, with HOPE-3 participants receiving stable background corticosteroid therapy. The studied population was aged at least ten years and late ambulatory or non-ambulatory. The sponsor’s refined proposed indication focuses on upper limb function. The evidence therefore concerns treatment added to existing care in this trial population; it does not establish an approved treatment position or support replacing corticosteroids or multidisciplinary care.
Heterogeneity of treatment effect
HOPE-3: cardiomyopathy subgroup analysis
FDA assessment of the cardiomyopathy definition
Formal treatment-by-subgroup interaction analysis
No evidence found.
Care management intervention strategies
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.