Givlaari, with the generic name givosiran, is a prescription medication used for the treatment of acute hepatic porphyria (AHP) in adults. Acute hepatic porphyria is a group of rare genetic disorders characterized by the buildup of certain chemicals called porphyrins, which can cause a range of symptoms, including severe abdominal pain, neurological issues, and potentially life-threatening attacks. Givlaari is a specialized therapy designed to reduce the frequency and severity of these acute attacks. Here is a detailed description of Givlaari:

Drug Class: Givlaari is classified as an RNA interference (RNAi) therapeutic. It works by reducing the production of a specific enzyme called delta-aminolevulinic acid synthase 1 (ALAS1) in the liver, which is involved in the synthesis of porphyrins. By inhibiting ALAS1, Givlaari helps to decrease the accumulation of porphyrins and prevent acute attacks.

Indications: Givlaari is indicated for the treatment of adults with acute hepatic porphyria (AHP), which includes several subtypes of porphyria characterized by acute attacks and symptoms related to the buildup of porphyrins in the liver.

Mechanism of Action: Givosiran works by utilizing RNA interference (RNAi) technology to inhibit the production of the ALAS1 enzyme in the liver. ALAS1 is responsible for the first step in the synthesis of porphyrins, and by reducing its activity, Givlaari helps to lower the levels of porphyrins in the body.

Dosage and Administration: The dosage of Givlaari is determined by a healthcare provider based on the individual patient’s weight. It is administered as a subcutaneous injection once a month. Treatment should be initiated and supervised by healthcare professionals experienced in the management of acute hepatic porphyria.

Effectiveness: Clinical trials have shown that Givlaari can significantly reduce the frequency of acute porphyria attacks and improve the quality of life for individuals with AHP. It helps to alleviate symptoms and reduce the need for hospitalization during acute attacks.

Side Effects: Like all medications, Givlaari may have side effects. Common side effects include injection site reactions, nausea, and fatigue. Patients should be monitored for adverse reactions, and any unusual symptoms should be reported to a healthcare provider.

Contraindications: Givlaari is not recommended for use in individuals with a known hypersensitivity to givosiran or any of its components.

Consultation with a Healthcare Provider: Before starting Givlaari, individuals should consult with their healthcare provider to determine if it is a suitable treatment option and to discuss the potential benefits and risks. Regular monitoring and follow-up with a healthcare provider are essential during Givlaari therapy to assess treatment effectiveness and manage potential side effects.

Givlaari represents a significant advancement in the management of acute hepatic porphyria (AHP), offering a targeted approach to reducing the frequency and severity of acute attacks. Its use should always be under the guidance and supervision of qualified healthcare professionals who can assess the individual’s specific medical condition and needs.