Givlaari

Givosiran

• Brand(s): Givlaari
• Dosage Form/Strength: 189 mg/mL injection
• Effective date: September 7, 2023

Initiation Criteria:

As treatment for the prevention of acute hepatic porphyria (AHP) attacks in patients meeting all the following criteria:

1. 18 years of age or older;
   AND
2. A confirmed diagnosis of acute hepatic porphyria (see note 1);
   AND
3. Has experienced four (4) or more porphyria attacks in the year prior to initiation of givosiran with each attack resulting in either hospitalization, an urgent healthcare visit, or treatment with intravenous hemin ;
   AND
4. Patient is under the care of a clinician experienced in the management of AHP.

Exclusion Criteria:

Givosiran will not be funded if it is used in combination with prophylactic IV hemin.

Renewal Criteria:

Renewals will be considered in those demonstrating a reduction in the annualized porphyria attack rate that required hospitalization, an urgent care visit, or acute treatment with IV hemin compared to the baseline period prior to start of givosarin AND who have not developed any unacceptable toxicities from treatment with givosiran.

Renewal requests must include the total number of porphyria attacks during the 12 month period of givosiran administration, including the dates of any attacks requiring hospitalization, urgent care visits, or acute IV hemin treatment.

Additionally, reconfirm that the patient does not meet exclusion criteria.

Notes:

1. The EAP request application must include at least one of or both (if available) of the following:
   i) documented biochemical test results, including one of, or both if they are available, urinary delta-aminolevulinic acid (ALA) and urinary porphobilinogen (PBG) tests, confirming the diagnosis of AHP, including the specified subtype, if it is known;
   ii) genetic test results confirming the diagnosis of AHP.
2. The baseline number of attacks before initiation of givosiran will be annualized. Requests should include the number of porphyria attacks over the specific period of time within the year before the initiation of givosiran and should include the approximate dates and the management of each attack (i.e. hospitalization, urgent care visit, dose of IV hemin used to treat the attack) for at least 4 of the attacks that occurred in the 12 month period before treatment initiation with givosiran. For patients who have already initiated givosiran therapy, this information for at least 4 attacks from the year prior to treatment initiation will also be required. The baseline attack history will also be required to administer renewal criteria.
3. If a patient experiences an acute attack while on givosiran, IV hemin as treatment for the acute attack may be administered as appropriate.
4. Patients with stable disease who transition to menopause may consider a trial of treatment discontinuation.
5. Retreatment will be considered in patients who experience a relapse of attacks following treatment discontinuation of givosarin.

Recommended Dose: 2.5 mg/kg SC injection monthly.
(Refer to the product monograph for dosing information)

Approval duration of initials and renewals: 1 year

EAP Drug Request Form:

Standard Form for EAP Drug Requests