Subcutaneous RNAi therapy has reached all phase 3 clinical endpoints and a new drug application has been submitted

Today, Alnylam Pharmaceuticals announced that vutrisiran, a subcutaneous RNAi therapy developed by the company, has reached the primary endpoint and all secondary endpoints in a phase 3 clinical trial for the treatment of patients with hereditary transthyretin (hATTR) amyloidosis.

Hereditary transthyretin amyloidosis is a hereditary and progressive disease caused by mutations in the TTR gene.

Vutrisiran is a subcutaneously administered RNAi therapy that can target and silence specific mRNAs, thereby blocking the production of wild-type and mutant transthyretin, and delaying disease progression.

▲Introduction of Onpattro and vutrisiran (picture source: Alnylam official website)

In the phase 3 clinical study called HELIOS-A, a total of 164 patients with hATTR amyloidosis received vutrisiran or Onpattro treatment.

In terms of Norfolk QoL-DN score, which assesses the quality of life of patients, patients with vutrisiran have improved 16.

Reference materials:

[1] Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran.

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