The Ministery of Food and Drug Safety said on the 26th that it approved Givlaari (ingredient name givosiran sodium), an imported orphan drug used to treat adults with acute hepatic porphyria (AHP).
Acute hepatic porphyria is a hereditary rare disease that occurs when enzymes needed for the synthesis of heme—an essential substance for oxygen transport in the body—in the liver are deficient, causing toxic intermediates such as aminolevulinic acid and porphobilinogen to accumulate. Patients experience severe abdominal pain and nerve damage.
Givlaari degrades the messenger ribonucleic acid (mRNA) for 5-aminolevulinate synthase 1 (ALAS1), an enzyme used in the heme synthesis process in the liver. This suppresses the production of the neurotoxic intermediates aminolevulinic acid and porphobilinogen, reducing the symptoms of acute hepatic porphyria.
Givlaari was developed by Alnylam Pharmaceuticals and received approval from the U.S. Food and Drug Administration (FDA) in 2019.
An official at the Ministery of Food and Drug Safety said, "We expect this drug to offer a new treatment opportunity to meet the unmet medical needs of adult patients with the rare disease acute hepatic porphyria."