Galsulfase

What it is and how it works

Mechanism of Action Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. MPS VI is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate, dermatan sulfate, throughout the body. This accumulation leads to widespread cellular, tissue, and organ dysfunction. NAGLAZYME is intended to provide an exogenous enzyme that will be taken up into lysosomes and increase the catabolism of GAG.

Indications

is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity. NAGLAZYME is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.

Dosage and administration (summary)

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. The recommended dosage is 1 mg per kg of body weight administered once weekly as an intravenous infusion. Recommendations Prior to NAGLAZYME Treatment Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis . Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment .

Contraindications

None. None.

Warnings and precautions

Immune-Mediated Reactions : Immune-mediated reactions can occur with NAGLAZYME. Monitor patients for the development of immune complex-mediated reactions while receiving NAGLAZYME. Risk of Acute Cardiorespiratory Failure : Caution should be exercised when administering NAGLAZYME to patients susceptible to fluid volume overload. Consider a decreased total infusion volume and infusion rate when administering NAGLAZYME to these patients. Appropriate medical monitoring and support measures should be available during infusion.

Pregnancy

Pregnancy Risk Summary Available data from a pregnancy sub-study within the MPS VI Clinical Surveillance Program and case reports with NAGLAZYME use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.

Storage and supply

injection is supplied as a sterile colorless to pale yellow, clear to slightly opalescent solution in clear Type I glass 5 mL vials, containing 5 mg galsulfase (expressed as protein content) per 5 mL solution. The closure consists of a siliconized chlorobutyl rubber stopper and an aluminum seal with a plastic flip-off cap.

Source: Galsulfase (Naglazyme) — US FDA prescribing information, 2024-09-18, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.