Inborn errors of metabolism:

Indications for GIVLAARI:

Acute hepatic porphyria (AHP).

Adult:

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.

Children:

Not established.

Warnings/Precautions:

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.

Pharmacologic Class:

Aminolevulinate synthase 1-directed small interfering RNA.

Interactions:

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.

Adverse Reactions:

Nausea, inj-site reactions, rash, serum creatinine increased, transaminase elevations, fatigue.

Generic Availability:

NO

How Supplied:

Single-dose vial—1