Givlaari (givosiran) — Medical Mutual

Initial criteria

• Patient is age ≥ 18 years; AND
• Patient will avoid known triggers of porphyria attacks (i.e., alcohol, smoking, exogenous hormones, hypocaloric diet/fasting, certain medications such as barbiturates, hydantoins, sulfa-antibiotics, anti-epileptics, etc.); AND
• Patient has not had or is not anticipating a liver transplant; AND
• Patient has a definitive diagnosis of acute hepatic porphyria as evidenced by one of the following: elevated urinary or plasma PBG (porphobilinogen) and ALA (delta-aminolevulinic acid) levels within the previous year; OR mutation in an affected gene as identified on molecular genetic testing; AND
• Patient has a history of at least two documented porphyria attacks (requirement of hospitalization, urgent healthcare visit or intravenous administration of hemin) OR one severe attack with CNS involvement (e.g., hallucinations, seizures, etc.) during the previous six months; AND
• Patients currently receiving prophylactic intravenous hemin therapy will discontinue hemin within 3 to 6 months following initiation of givosiran

Reauthorization criteria

• Patient continues to meet the universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc.; AND
• Absence of unacceptable toxicity from the drug (e.g., anaphylactic reactions, severe hepatic toxicity, severe renal toxicity, severe injection site reactions, increase in blood homocysteine levels, acute pancreatitis); AND
• Disease response as evidenced by a decrease in the frequency of acute porphyria attacks, and/or hospitalizations/urgent care visits, and/or a decreased requirement of hemin intravenous infusions for acute attacks; AND
• Patient has a reduction/normalization of biochemical markers (i.e., ALA, PBG) compared to baseline; AND
• Patient will not use in combination with prophylactic intravenous hemin therapy

Approval duration

initial 6 months, renewal 12 months