Elfabrio is a pegylated enzyme replacement therapy (pegunigalsidase alfa-iwxj) that may be used to treat adults with confirmed Fabry disease. The correct technical term for Elfabrio is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.

Fabry disease is a rare, inherited, X–linked, disorder caused by a deficiency of the alpha-galactosidase-A enzyme. People with Fabry’s disease do not have enough alpha-galactosidase-A to break down a fatty substance called globotriaosylceramide (Gb3) into smaller components that the body can use for energy. As a result, Gb3 accumulates in blood vessels and tissue, increasing the risk of a heart attack, stroke, or kidney failure. Symptoms may include numbness or tingling in the hands or feet, extreme pain during physical activity, heat or cold intolerance, dizziness, or flu-like symptoms. Males tend to have more severe symptoms. Fabry disease is also known as alpha-galactosidase-A enzyme deficiency or Anderson-Fabry disease.

Elfabrio replaces the missing alpha-galactosidase A enzyme, allowing the body to break down Gb3 and prevent its accumulation.

Elfabrio was approved on May 9, 2023.

Warnings and precautions include infusion-associated reactions (IARs) and membranoproliferative glomerulonephritis.

If severe IARs occur, discontinue Elfabrio and initiate appropriate medical treatment.

Monitor serum creatinine and urinary protein to creatinine ratio should be monitored and Elfabrio discontinued if glomerulonephritis is suspected until a diagnostic evaluation can be conducted.

Before taking this medicine

Consider pre-treatment with antihistamines, antipyretics, and/or corticosteroids.

Pregnancy

There is no human data regarding the use of Elfabrio during pregnancy but because it is an enzyme replacement treatment it is not expected to cause any adverse outcomes. Animal reproduction studies have reported no adverse effects.

If a patient becomes pregnant while receiving Elfabrio, healthcare providers should report the exposure to the pregnancy safety study by calling 1-888-661-9260.

Lactation

There is no data regarding the effects of Elfabrio on a breastfeeding infant or on milk production.

Children

The safety of Elfabrio in children has not been established.

Elfabrio is administered as an intravenous infusion by a healthcare provider every two weeks.

The recommended dosage is 1 mg/kg every 2 weeks.

Pretreatment with antihistamines, antipyretics, and/or corticosteroids may be considered to reduce the risk of infusion-related reactions and you will be monitored for one to two hours after the infusion.

The Elfabrio product label carries a Boxed Warning for hypersensitivity reactions, including anaphylaxis.

Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, Elfabrio should be discontinued immediately and appropriate medical treatment initiated.

Common adverse reactions include infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in the hands and feet, and sinusitis.

Interaction studies have not been conducted with Elfabrio.