Givlaari (givosiran) is a subcutaneous injection given just under the skin that is prescribed to treat adults with acute hepatic porphyria (AHP).
AHP is a family of very rare genetic diseases that result in chronic illness with symptoms that include stomach pain, nausea and vomiting, muscle weakness, seizures, confusion, hallucinations, and skin lesions, ulcers, or erosions from sun exposure.
Due to its vague symptoms, AHP can be difficult to diagnose and is potentially fatal if not treated properly. People with AHP have an overproduction of the protein delta-aminolevulinate synthase 1 (ALAS1) in the liver. ALAS1 regulates the heme biosynthesis pathway in the liver that produces the body’s blood properties and liver functions. When ALAS1 is active, it creates the byproducts aminolaevulinic acid (ALA) and porphobilinogen (PBG), which are toxic in large quantities. When ALAS1 is not being regulated in people with AHP, it is overproduced, releasing toxic amounts of ALA and PBG into the body. ALA and PBG have been linked with AHP symptoms and flares.
Givlaari specifically targets the ALAS1 protein mRNA to decrease its production and ultimately decrease ALA and PBG release. This helps decrease AHP symptoms and flares.
Other side effects:
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Once per month
Before your injection, it is important to drink plenty of water and come to your appointment hydrated. Additionally, you must alert us as soon as possible to any changes in your insurance to ensure that we are still an in-network provider for your infusion therapy.