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MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › Other drugs for disorders of the musculo-skeletal system
delandistrogene moxeparvovec
M09AX15
Forms & Strengths
- Intravenous suspension: 1 x 1^14 vector genomes per milliliter (vg/mL)
Adult Dosing
- Not applicable; indicated for pediatric populations with Duchenne muscular dystrophy
Pediatric Dosing
- 1.33 x 10^14 vector genomes per kilogram (vg/kg) of body weight administered as a single intravenous infusion
Indications
- Treatment of Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 years with a confirmed mutation in the DMD gene
Mechanism of Action
- Recombinant adeno-associated virus serotype rh74 (AAVrh74) vector-based gene therapy designed to deliver a transgene encoding a shortened, functional dystrophin protein (micro-dystrophin) to skeletal and cardiac muscle
Contraindications
- Any deletion in the DMD gene spanning exons 8 and/or 9 due to risk of severe immune-mediated myositis
- Pre-existing neutralizing antibodies to AAVrh74 higher than the threshold established by clinical screening
- Previous severe hypersensitivity to prednisolone or other systemic corticosteroids
Adverse Reactions
- Vomiting
- Nausea
- Pyrexia
- Elevated transaminases (alanine aminotransferase and aspartate aminotransferase increase)
- Immune-mediated myositis
- Myocarditis
- Thrombocytopenia
Drug Interactions
- Concomitant immunomodulatory or immunosuppressive agents may affect the immune response to the AAVrh74 vector
- Live vaccines should be delayed until systemic corticosteroid therapy is successfully tapered and discontinued following gene therapy
Curated Content: Needs Vetting Before Put to Clinical Use