Givlaari™

Drug - Givlaari™ (givosiran) [Alnylam Pharmaceuticals Inc.]

April 2020

Therapeutic area - Acute hepatic porphyria

Initial approval criteria

Patient must be ≥ 18 years or older AND
Patient has a definitive diagnosis of acute hepatic porphyria, including acute intermittent porphyria, variegate porphyria, hereditary coproporphyria, or ALA dehydratase deficient porphyria, as evidenced by one of the following:
- patient has had elevated urinary or plasma PBG and ALA levels within the previous year OR
- patient has a mutation in an affected gene as identified on molecular genetic testing AND
Patient has a history of ≥ 2 documented porphyria attacks (i.e., requirement of hospitalization, urgent healthcare visit, or IV administration of hemin) OR 1 severe attack with central nervous system involvement (e.g., hallucinations, seizures, etc.) during the previous 6 months AND
Patient will avoid concomitant use with, or use decreased doses of, CYP1A2 or CYP2D6 substrates, for which minimal concentration changes may lead to serious or life-threatening toxicities (e.g., clozapine, amitriptyline, theophylline, verapamil, clomipramine, clonidine, etc.) AND
Patient will avoid known triggers of porphyria attacks (e.g., alcohol, smoking, exogenous hormones, hypocaloric diet/fasting, certain medications such as barbiturates, hydantoins, sulfa-antibiotics, anti-epileptics, etc.) AND
Patients currently receiving prophylactic IV hemin therapy will discontinue hemin within 3 to 6 months of initiation with givosiran AND
Patient has NOT had or is anticipating a liver transplant
Initial approval is for 6 months

Patient must continue to meet the above criteria AND
Patient must demonstrate disease response as evidenced by a decrease in the frequency of acute porphyria attacks, and/or hospitalizations/urgent care visits, and/or a decrease requirement of hemin IV infusions AND
Patient has a reduction/normalization of biochemical markers (e.g., ALA, PBG) compared to baseline AND
Patient will NOT use in combination with prophylactic IV hemin therapy AND
Patient has NOT experienced any treatment-restricting adverse effects (e.g., anaphylactic reactions, severe hepatic toxicity, severe renal toxicity, severe injection site reactions, etc.) AND
Patient has NOT received a liver transplant
Renewal approval is for 12 months

Givlaari must be billed as a medical claim.

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