EARLY ACCESS PROGRAM FOR GIVOSIRAN

Givosiran is a subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP). Where it is approved, it is marketed as GIVLAARI® (givosiran).

Eligibility Requirements

To be eligible to seek access to givosiran through the early access program (EAP), participants must meet certain requirements, including, but not limited to:

 

  • Have a confirmed diagnosis of acute hepatic porphyria (AHP), including acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), variegate porphyria (VP), or ALAD-deficient porphyria (ADP)
  • Are 12 years of age or older
  • Are not currently enrolled in an ongoing interventional givosiran clinical trial
  • Have no known serious comorbidities, or any of the following laboratory parameter assessments at screening (including elevated liver enzymes), and must be deemed to be fit for the program by treating physician, specifically:
    • Alanine aminotransferase (ALT) >2×ULN
    • Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2×ULN
    • Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2 using the Modification of Diet in Renal Disease (MDRD) formula or the Schwartz Bedside Formula
    • On an active liver transplantation waiting list

 

If you are a patient interested in participating, please speak with your physician about the potential benefits and risks of taking part in the program.

 

If you are a patient living in a country where givosiran is not commercially available and are interested in seeking access to givosiran through the EAP, please speak with your physician. Alnylam can only respond to a physician requesting access to givosiran on behalf of a patient in his/her care.

 

If you are a physician who is seeking to obtain access to givosiran for a patient in your care, please contact managedaccess@clinigengroup.com

 

If you are a physician or patient and have a medical question, please contact medinfo@alnylam.com.

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