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elapegademase

L03AX21

Forms & Strengths

Injection: 2.4 mg/2 mL (1.2 mg/mL) in single-dose vials

Adult Dosing

Initial treatment-naive patients: 10 mcg/kg once weekly intramuscularly
Switching from pegademase bovine: Transition based on equivalent adenosine deaminase (ADA) activity and clinical response, typically maintaining weekly dosing

Pediatric Dosing

Neonates and pediatric patients: 10 mcg/kg once weekly intramuscularly
Maintenance titration: Adjust dose based on trough plasma ADA activity levels (> 15 micromol/hr/mL) and trough deoxyadenosine nucleotide (dAXP) levels in erythrocytes (< 0.02 micromol/mmol)

Indications

Enzyme replacement therapy for treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID) in pediatric and adult patients

Mechanism of Action

Recombinant adenosine deaminase (ADA) conjugated to polyethylene glycol (PEG)
Catalyzes the conversion of adenosine and deoxyadenosine to inosine and deoxyinosine, respectively
Reduces toxic intracellular accumulation of deoxyadenosine nucleotides (dAXP) which otherwise impair lymphocyte proliferation and function in ADA-SCID

Contraindications

Severe hypersensitivity to elapegademase or its components, unless no other therapeutic options exist and desensitization is performed
Active, untreated severe infections (stabilize prior to initiation)

Adverse Reactions

Injection site reactions (pain, erythema)
Headache
Cough
Arthralgia
Development of neutralizing anti-drug antibodies
Thrombocytopenia (rare)

Drug Interactions

Live vaccines: Avoid administration of live virus vaccines in immunodeficient patients receiving enzyme replacement therapy
Immunosuppressive agents: May reduce the immune reconstitution efficacy of elapegademase

Curated Content: Needs Vetting Before Put to Clinical Use