EARLY ACCESS PROGRAM FOR LUMASIRAN

Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1). The safety and efficacy of lumasiran have not been evaluated or approved by health authorities in any country.

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:

  • A documented diagnosis of PH1
  • Age 6 or older
  • Relatively preserved renal function (estimated glomerular filtration rate [eGFR] greater than 45 mL/min/1.73 m2) at screening
  • Meet the requirements for urine oxalate excretion
  • No clinically significant health concerns (with the exception of PH1)
  • No clinically significant abnormal laboratory results at screening
  • Have not received an investigational agent within 30 days before the first dose of lumasiran or are not in follow-up of another clinical study
  • Have not previously or are not currently participating in a lumasiran clinical study
  • No history of renal or liver transplant
  • Not currently pregnant, planning a pregnancy, or breast-feeding or are of child-bearing potential and willing to use contraception

 

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.

 

In the U.S., additional information can be found by visiting ClinicalTrials.gov/NCT04125472.

 

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

 

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

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