FDA approved the third RNAi drug in three years

FDA approved the third RNAi drug in three years

After being approved by the European Union last week, Alnylam pharmaceuticals announced today that its RNAi therapy oxlumo (lumasiran) was approved by the US FDA for the treatment of primary hyperoxaluria type 1 (Ph1). This is the third RNAi treatment approved by FDA within three years, and the 44th treatment approved by FDA this year.

Ph1 is a rare hereditary liver disease. The mutation of the gene encoding human alanine glyoxylate aminotransferase (AGT) leads to the deletion of AGT. This will cause excessive oxalate production in the body of patients, which will accumulate in the kidney and can not be discharged in time. Severe Ph1 patients have oxalate deposition in multiple parts of the body, which damages the kidney and related organs. Liver and kidney transplantation are often required.

Lumasiran is a subcutaneous RNAi therapy that targets the mRNA of hao1 gene encoding glycolate oxidase (go) in the liver. By reducing the expression of go, the production of oxalate in liver can be reduced. This 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 liver.

In June this year, Alnylam released the data of lumasiran's key phase 3 clinical trial. In the illuminate-a clinical trial, 39 patients with Ph1 received either lumasiran or placebo. During the 3-6 months after treatment, the mean urinary oxalic acid level in the lumasiran group decreased by 65.4% compared with baseline, and decreased by 53.5% compared with the placebo group. In the lumasiran group, urinary oxalic acid levels returned to normal in more than half of the patients, and nearly normal in 84% of the patients, compared with 0% in the placebo group.

In a press release, Dr. Akshay vaishnaw, President of research and development of Alnylam, pointed out in a press release that the approval of oxlumo shows that its RNAi platform is extremely high-yielding and can bring innovative therapies to more patients. As the first targeted therapy for Ph1, its significance for patients and their families is self-evident.