Pegzilarginase

LOARGYS is indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction. This indication is approved under accelerated approval based on reduction of plasma arginine. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. LOARGYS is an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.

ARG1-D is an inherited metabolic disease characterized by deficiency of the arginase 1 enzyme and associated with the persistent elevation of plasma arginine. LOARGYS provides an exogenous source of the deficient human arginase 1 enzyme activity in patients with ARG1-D and reduces plasma arginine by converting it to urea and ornithine.

• Administer LOARGYS under the supervision of a health care provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. • Initiate LOARGYS in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. • Consider pre‑medication with antihistamines. • Obtain a baseline plasma arginine concentration prior to initiating treatment. • Recommended starting dosage of LOARGYS is 0.1 mg/kg administered by intravenous infusion once weekly. • Maximum recommended dosage is 0.2 mg/kg once weekly. • See the Full Prescribing Information for recommended titration and maintenance dosage and recommended plasma arginine level testing during treatment.

None. None.

Boxed warning (hypersensitivity reactions including anaphylaxis): Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Hypersensitivity: If a severe hypersensitivity reaction occurs, discontinue LOARGYS and immediately initiate appropriate medical treatment, including epinephrine.

The following adverse reactions are described below and elsewhere in the labeling: • Hypersensitivity Reactions. Most common adverse reactions (>10%) are vomiting, pyrexia, infusion associated reactions and constipation.

Store refrigerated at 2 °C to 8 °C (36 °F to 46 °F) in the original carton to protect from light. Do not freeze. Do not shake.