Section 3 of 6
Product information and disease description
63 evidence topics · 50 sources
Product description
Phase of product development
Orphan drug and rare pediatric disease designations
Breakthrough Therapy designation
Priority Review and FDA action date
Launch: Anticipated in the first quarter of 2027
FDA Advisory Committee meeting date
No evidence found.
Product information
Generic, brand name and therapeutic class of product
Dosage forms and strengths
Investigational formulation and recorded strengths
| Registry field | Quoted record |
|---|---|
| Pharmaceutical form | “Solution for infusion” |
| First recorded strength, mg | “200” |
| Second recorded strength, mg | “350” |
Commercial formulation, packaging, and storage requirements
No evidence found.
Material safety data sheet
No evidence found.
Average sales price and wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
Therapeutic approach
AHFS classification code
No evidence found.
Indication
Pharmacology
Mechanism of action
Pharmacodynamics
FORCE platform toxicology: repeat-dose studies in cynomolgus monkeys
FORCE-M23D and CNS outcomes: hTfR1/mdx mice
FORCE-M23D muscle pathology: D2-mdx mouse model
DYNE-251 preclinical exon skipping: nonhuman primates
DELIVER: dose-dependent target engagement
Pharmacokinetics
Muscle drug concentration, initial DELIVER data
Plasma exposure, clearance, half-life, and accumulation
No evidence found.
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
DELIVER: non-ambulatory participant eligibility
Renal impairment, hepatic impairment, pregnancy, and lactation
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on Zeleciment rostudirsen
Drug–drug, drug–food, and drug–disease interactions
No evidence found.
Effects of Zeleciment rostudirsen on other drugs
No evidence found.
Dosing and administration
Dosage
Proposed dose, route, and frequency
Administration
Investigational route of administration
Final administration-site requirements
No evidence found.
Access and distribution
Commercial distribution strategy
No evidence found.
Commercial patient support program
No evidence found.
Co-prescribed/Concomitant therapies
DELIVER: concomitant glucocorticoid requirement
Effect of Zeleciment rostudirsen on quality measures
Z-rostudirsen-specific effect on quality measures
No evidence found.
Evaluation of care-consideration implementation
Product comparison
Treatment alternatives: eteplirsen prescribing information
| Product-information element | Direct quotation |
|---|---|
| Mutation requirement | “amenable to exon 51 skipping” |
| Dose | “30 milligrams per kilogram of body weight once weekly” |
| Administration | “intravenous infusion over 35 to 60 minutes” |
Head-to-head comparison with an approved therapy
No evidence found.
Place of product in therapy
Disease description
Definition and etiology
Epidemiology
Incidence of Duchenne muscular dystrophy
Prevalence of Duchenne muscular dystrophy
Natural history, survival, and mortality
Genotype and loss of ambulation in corticosteroid-treated patients
Survival in more recent birth cohorts
Pathophysiology
Diagnosis
Diagnostic delay in males without a family history of muscular dystrophy
Clinical presentation - signs and symptoms
Motor symptoms: early weakness and developmental delay
Long-term morbidity
Burden of Duchenne muscular dystrophy
Humanistic burden and health-related quality of life
Economic burden and healthcare resource utilization
U.S. healthcare costs: mean total healthcare costs, DMD versus non-MD
Economic impact of Duchenne muscular dystrophy on families
Annual productivity-cost estimates
Economic impact of diagnostic testing
Product-specific economic effect of eligibility testing
No evidence found.
Approaches to treatment
Current treatment options and standard of care
Corticosteroids
Exon-skipping antisense oligonucleotides
Eteplirsen therapeutic class
Histone deacetylase inhibitors
Givinostat therapeutic class
Gene therapy
Treatment alternatives: delandistrogene moxeparvovec-rokl
Rehabilitation and orthopaedic care
Cardiac therapies
Respiratory support
Bone health and endocrine care
Psychosocial and transition support
Limitations of current therapies
Summary
Available treatments have different eligibility restrictions, administration requirements, and evidentiary limitations. Eteplirsen is restricted to exon 51-skipping-amenable mutations and requires weekly intravenous infusion; its labeling states that continued approval may depend on verification of clinical benefit. Delandistrogene moxeparvovec-rokl is limited to ambulatory individuals aged four years or older and carries a boxed warning for serious liver injury and acute liver failure, including fatal outcomes. The 2019 ICER assessment of eteplirsen and golodirsen is historical and is not an assessment of z-rostudirsen.
Eteplirsen confirmatory-evidence requirement
Place in treatment, anticipated use, and care setting
Summary
Zeleciment rostudirsen is being developed for individuals with DMD mutations amenable to exon 51 skipping. The proposed regimen is 20 mg/kg intravenously every four weeks, with doses expressed as the PMO component. DELIVER required stable background glucocorticoids for at least 12 weeks. The reported commercial launch remains conditional on approval; final commercial distribution and administration-site requirements are not established in this report.
Heterogeneity of treatment effect
Care management intervention strategies
Clinical-trial participation support
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.