ELFABRIO

What it is

Elfabrio contains pegunigalsidase alfa, 20 mg in a 10 mL vial (2 mg/mL). It is a concentrated injection. Pegunigalsidase alfa is a pegylated recombinant form of human alpha-galactosidase-A. Pegunigalsidase alfa supplements or replaces alpha-galactosidase-A, the enzyme that catalyses the hydrolysis of the terminal α-galactosyl moieties of oligosaccharides and polysaccharides in the lysosome, where it is thought to exert enzymatic activity and reduce accumulated globotriaosylceramide (Gb3). Elfabrio is subject to additional monitoring in Australia.

Approved indications

— Long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease.

Dosing overview

The recommended dose of pegunigalsidase alfa is 1 mg/kg of body weight administered once every two weeks. Elfabrio is for intravenous infusion use only. For initial infusions, the infusion duration should be not less than 3 hours, with infusion rates ranging from 0.83 to 2.78 mL/min depending on body weight. For maintenance infusions, the target infusion duration can be reduced based on patient's tolerability to not less than 1.5 hours. No dose adjustment is needed in patients with renal or hepatic impairment.

Key safety warnings

Infusion-related reactions (IRRs) are defined as any related adverse events with onset after start of infusion and up to 2 hours after end of infusion. The most commonly observed symptoms of IRRs were hypersensitivity, itching, nausea, dizziness, chills and muscular pain. The management of IRRs must be based on the severity of the reaction, and include slowing the infusion rate and treatment with medicinal products such as antihistamines, antipyretics and/or corticosteroids, for mild to moderate reactions. The presence of anti-drug antibodies (ADAs) to Elfabrio may be associated with a higher risk of IRRs, and severe IRRs are more likely to occur in ADA positive patients. Allergic-type hypersensitivity reactions may manifest and can include localised angioedema (including swelling of the face, mouth, and throat), bronchospasm, hypotension, generalised urticaria, dysphagia, rash, dyspnoea, flushing, chest discomfort, pruritus, and nasal congestion. If a severe allergic or anaphylactic-type reactions occur, immediate discontinuation of Elfabrio is recommended and current medical standards for emergency treatment should be followed. Immune complex depositions can potentially occur during treatment with enzyme replacement therapies as a manifestation of immunological response to the product. A single case of glomerulonephritis membranoproliferative was reported during the clinical development of Elfabrio, due to immune depositions in the kidney. It is recommended to monitor serum creatinine and urinary protein to creatinine ratio. Discontinue Elfabrio if glomerulonephritis is suspected, until a diagnostic evaluation can be conducted.

Contraindications

Elfabrio is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients.

PBS listing

Elfabrio was recommended by PBAC in July 2025 for Fabry disease. It was recommended on a cost-minimisation basis to migalastat. Further details regarding strength, item count, restriction type, and ex-manufacturer price are not provided in the available source documents.

Regulatory history

Elfabrio pegunigalsidase alfa 20 mg/10 mL concentrated injection vial was first listed on the ARTG on 26 May 2025. The AusPAR approval date was 12 May 2025. The PBAC recommended Elfabrio in July 2025 for long-term enzyme replacement therapy in adult patients with Fabry disease.