Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1)—the gene encoding glycolate oxidase (GO)—for the treatment of primary hyperoxaluria type 1 (PH1). Where it is approved, it is marketed as OXLUMO® (lumasiran).

Eligibility Requirements

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

  • Of any age and with a documented diagnosis of PH1.
  • Not currently enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives, whichever is longer, since ending another investigational device or drug study or receiving other investigational agent(s)
  • Not previously dosed with lumasiran in a clinical study.
  • No history of liver transplant.
  • No medical conditions or comorbidities that, in the opinion of the physician, would interfere with program compliance or the patient’s safety.

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

If you are a physician who is seeking to obtain access to lumasiran for a patient in your care, please contact eap@alnylam.com.

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


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