A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of Givosiran (ALN-AS1) for the Treatment of Acute Hepatic Porphyrias (AHP)

Trial Profile

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of Givosiran (ALN-AS1) for the Treatment of Acute Hepatic Porphyrias (AHP)

Planning
Phase of Trial: Phase III

Latest Information Update: 07 Sep 2017

At a glance

  • Drugs Givosiran (Primary)
  • Indications Acute intermittent porphyria; Liver disorders; Porphyria
  • Focus Registrational; Therapeutic Use
  • Sponsors Alnylam Pharmaceuticals
  • Most Recent Events

    • 07 Sep 2017 According to an Alnylam Pharmaceuticals media release, the company expects interim analysis data in mid-2018.
    • 07 Sep 2017 According to an Alnylam Pharmaceuticals media release, based on the new givosiran phase 3 design, the company is now guiding that pending FDA review of the program at the time of interim analysis and assuming positive results, it expects to submit an NDA at or around year-end 2018.
    • 07 Sep 2017 According to an Alnylam Pharmaceuticals media release, the company has reached alignment with the U.S.FDA on a phase 3 study design which includes an interim analysis based on reduction of a urinary biomarker, aminolevulinic acid, as a surrogate endpoint that the FDA considers to be reasonably likely to predict clinical benefit.
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