Inborn errors of metabolism:
Indications for GIVLAARI:
Acute hepatic porphyria (AHP).
Give by SC inj into the abdomen, the back or side of the upper arms, or the thighs; rotate inj sites. 2.5mg/kg once monthly. For severe or clinically significant transaminase elevations, reduce dose to 1.25mg/kg once monthly; if no recurrence, may increase to 2.5mg/kg.
Have medical support readily available. Monitor for anaphylaxis; discontinue immediately and treat if occurs. Obtain LFTs prior to initiation, repeat monthly during the 1st 6 months, then as clinically indicated. Interrupt or discontinue therapy if clinically significant transaminase elevations occur (see Adults). Monitor renal function. Pregnancy. Nursing mothers.
Aminolevulinate synthase 1-directed small interfering RNA.
Avoid concomitant use with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities; if unavoidable, decrease the dose of substrates.
Nausea, inj-site reactions, rash, serum creatinine increased, transaminase elevations, fatigue.