EARLY ACCESS PROGRAM FOR GIVOSIRAN

Givosiran is an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP. The safety and efficacy of givosiran have not been evaluated or approved by health authorities in any country.

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
  • Patients must have experienced a porphyria attack and be considered by their treating physician to be at risk for having an attack in the future
  • Have adequate venous access for program sample collections as judged by the Investigator for study sample collections
  • Have not previously participated in, or are currently enrolled in, an ongoing interventional givosiran clinical trial

    • Patients who received placebo and withdrew from the ENVISION Phase 3 study may be eligible subject to local requirements and the complete EAP eligibility requirements
  • Have no known serious comorbidities, or any of the following laboratory parameter assessments at screening (including elevated liver enzymes), and must 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) <30 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.

 

The givosiran early access program is currently open in select countries in North America, Europe, and Asia. In the U.S., additional information can be found by visiting ClinicalTrials.gov/NCT04056481.

 

Only a licensed physician can request givosiran. If you are a patient who is interested in seeking access to givosiran through the early access program, please speak to 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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