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alipogene tiparvovec

C10AX10

Forms & Strengths

AAV1-LPL(S447X) suspension for intramuscular injection
Concentration: 3 x 10^12 genomic particles (gc)/mL

Adult Dosing

Single administration via multiple intramuscular injections
Total calculated dose based on body weight: 1 x 10^14 gc/kg

Pediatric Dosing

Safety and efficacy have not been established in pediatric patients
Not recommended for use in patients under 18 years of age

Indications

Treatment of adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD)
Indicated for patients suffering from pancreatitis attacks

Mechanism of Action

Recombinant adeno-associated virus type 1 (AAV1) vector containing the human lipoprotein lipase gene variant LPL(S447X)
Facilitates transfer and local expression of the LPL enzyme in skeletal muscle cells
Reduces chylomicronemia and lowers plasma triglyceride levels in deficient patients

Contraindications

History of severe hypersensitivity to the active substance or any excipients
Presence of neutralizing antibodies against AAV1 viral capsid
Active systemic viral, bacterial, or fungal infections
Advanced hepatic disease or severe renal impairment

Adverse Reactions

Transient injection site pain and erythema
Elevated creatine phosphokinase (CPK)
Development of anti-AAV1 antibodies
Transient asymptomatic elevations in liver transaminases

Drug Interactions

Immunosuppressive agents (e.g., corticosteroids, cyclosporine) may alter immune response to the AAV1 vector and should be managed carefully
Concurrent use with hepatotoxic drugs may increase risk of liver function test abnormalities

Curated Content: Needs Vetting Before Put to Clinical Use