LOARGYS IS NOW APPROVED IN THE U.S. UNDER ACCELERATED APPROVAL!

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions Including Anaphylaxis: Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including LOARGYS. Hypersensitivity reactions that were mild to moderate in severity occurred in 13% (6/48) of LOARGYS-treated subjects in clinical trials. Hypersensitivity reactions have included facial swelling, rash, flushing and dyspnea. The reactions generally occurred with the first few doses but may occur later in treatment.

Administration of LOARGYS should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis in a healthcare setting with appropriate medical monitoring and support measures. Premedication with an antihistamine and/or corticosteroid should be considered in patients who previously have developed a hypersensitivity reaction. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LOARGYS and immediately initiate appropriate medical treatment, including use of epinephrine. Consider the risks and benefits of re-administering LOARGYS in patients who have experienced a severe hypersensitivity reaction. Caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions and to seek immediate medical attention should symptoms occur. If a mild or moderate reaction occurs, consider treatment with antihistamines and/or corticosteroids.

ADVERSE REACTIONS

The most common adverse reactions are vomiting, pyrexia, infusion associated reactions and constipation.

USE IN SPECIFIC POPULATIONS

Pregnancy: There are no available data on LOARGYS use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.

Lactation: There is no data on the presence of LOARGYS in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for LOARGYS and any potential adverse effects on the breast-fed infant from LOARGYS or from the underlying maternal condition.

Pediatric: The safety and effectiveness of LOARGYS have been established for the reduction of plasma arginine in pediatric patients 2 years and older with ARG-1 D, in conjunction with dietary protein restriction. The safety and effectiveness of LOARGYS have not been established for the reduction of plasma arginine in pediatric patients aged less than 2 years with ARG-1 D.

Geriatric: Clinical studies of LOARGYS did not include subjects 65 years of age and older to determine whether they respond differently from younger adult subjects.

INDICATION

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.

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.