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ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Enzymes
rebisufligene etisparvovec
A16AB29
Forms & Strengths
- Intravenous infusion suspension provided in single-dose vials
- Strength tailored based on patient body weight (viral vector genomes/kg)
Adult Dosing
- Administered as a single-dose intravenous infusion
- Exact dosage determined by clinical protocol and patient weight for gene therapy delivery
Pediatric Dosing
- Safety and efficacy established in pediatric patients per specific mucopolysaccharidosis indication guidelines
- Weight-based single intravenous infusion dose calculated by treating clinician
Indications
- Treatment of Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome)
Mechanism of Action
- Adeno-associated virus serotype-derived gene therapy vector delivering a functional copy of the ARSB gene
- Restores systemic production of N-acetylgalactosamine 4-sulfatase to reduce cellular accumulation of glycosaminoglycans (GAGs)
Contraindications
- Hypersensitivity to the active substance or any excipients
- Active systemic infections prior to completion of treatment course and immunosuppression stabilization
- Pre-existing neutralizing antibodies to the AAV vector above acceptable threshold (if specified by protocol)
Adverse Reactions
- Infusion-related reactions including pyrexia, chills, and headache
- Transient elevations in liver transaminases requiring immunomodulatory management
- Immune response directed against the viral vector or transgene product
Drug Interactions
- Concurrent use of immunosuppressive agents (e.g., corticosteroids) typically required around administration to manage immune response to viral vector
- Potential interference with other live vaccines due to immunosuppressive regimens
Curated Content: Needs Vetting Before Put to Clinical Use