The Ministery of Food and Drug Safety said on the 21st it approved the Fabry disease treatment "Elfabrio (ingredient name pegunigalsidase alfa)."
Fabry disease is a rare lysosomal storage disorder caused by a deficiency of the enzyme "alpha-galactosidase A" (alpha-galactosidase A) in the body. The enzyme breaks down glycolipids and glycoproteins in the lysosomes of cells. When the enzyme is deficient, glycolipids accumulate throughout the body, causing a variety of symptoms.
Representative symptoms include angiokeratoma of the skin, abdominal pain, proteinuria, pain in the fingertips and toes, hypohidrosis and anhidrosis in which sweat does not come out well, and hearing and vision impairment.
Elfabrio is a gene recombination-based enzyme replacement therapy developed by Italy's Chiesi Pharma. It received U.S. Food and Drug Administration (FDA) approval in 2023.
The drug works by supplementing the deficient alpha-galactosidase A enzyme in Fabry disease patients, reducing the accumulation of glycolipids in the body and alleviating disease progression. It is used for long-term enzyme replacement therapy in patients diagnosed with Fabry disease.
The Ministery of Food and Drug Safety said, "With this approval, treatment options for Fabry disease patients are expected to expand," and added, "We will continue efforts to swiftly supply new treatments to patients with rare diseases."