A third RNAi drug was approved by the European Union today

PH1 is a rare genetic liver disease in which a patient is missing AGT due to a mutation in the gene that encodes the human alanine-acetaldehyde amino transferase (AGT).
this can cause excessive amounts of herbal acid to be produced in the patient's body, which accumulates in the kidneys and cannot be discharged in time.
severe PH1 patients with hydrochloric acid deposits in multiple parts of the body, damaging the kidneys and related organs, often requiring liver and kidney transplants.
Lumasiran is a subsurfic injection of RNAi therapy that targets the mRNA of the HAO1 gene that encodes ethanol oxidase (glycolate oxidase, GO) in the liver.
reduces the production of herbic acid in the liver by reducing the expression of GO.
RNAi therapy uses Alnylam's Enhanced Stabilization Chemistry-GalNAc delivery technology platform.
this technology platform not only improves the stability of RNAi therapy, but also promotes the targeted delivery of therapy to the liver.
its application for a new drug to the FDA has been eligible for priority review and is expected to be answered no later than December 3 this year.
: Alnylam's official website, Alnylam, published data on a key Phase 3 clinical trial of lummasiran in June, in which 39 PH1 patients were treated with lumasiran or a placebo.
3-6 months after treatment, the average uric acid levels in the lumasiran group decreased by 65.4% compared to the baseline and by an average of 53.5% compared to the placebo group.
in the lumasiran group, uric acid levels returned to normal in more than half of patients, with 84 percent nearing normal levels, up from 0 percent in the placebo group.
: s1. Alnylam Receives Approval for OXLUMO™ (lumasiran) in The European Union for The Treatment of Primary Hyperoxaluria Type 1 in All Age Groups, Retrieved November 19, 2020, from