GIVOSIRAN INJ,SOLN
Clinical Criteria Summary
Exclusion Criteria
- History of or anticipated liver transplantation
Inclusion Criteria
- Provider/Specialty Restriction: Restricted to VA / VA Community Care provider in dermatology, gastroenterology, gynecology, hematology, hepatology, or neurology, or in consultation with one of these specialties
- Diagnosis Requirements: Diagnosis of acute hepatic porphyria by at least ONE of the following:
- Genetic testing confirming mutation for acute intermittent porphyria, hereditary coproporphyria, variegate porphyria, or aminolevulinic acid (ALA) dehydratase deficient porphyria
- OR confirmation of acute hepatic porphyria by documentation of clinical features (e.g., severe abdominal pain or other neurovisceral symptoms) and laboratory parameters (e.g., urinary or plasma porphobilinogen [PBG] or ALA at least four times the upper limit normal) during an acute attack
- Disease Activity & Attack History: Active disease with at least 2 porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous hemin administered within the past 6 months despite avoidance of potential precipitating factors AND intravenous hemin prophylaxis administered more than once weekly or documented intolerance to hemin
- Women with Menstrual Cycle-Related Attacks: Intolerance to or ineffective prophylaxis with a gonadotropin-releasing hormone analogue
Continuation & Evaluation Criteria
- Evaluate continuation of therapy based on patient response as evidenced by improvement (e.g., reduction in number of attacks requiring hospitalization, urgent care visit, or administration of intravenous hemin), disease stabilization or absence of disease progression, and ability to tolerate treatment