First siRNA drug to treat nerve disease

September 14, 2018 0 By CH Unnikrishnan

Alnylam Pharmaceuticals, Inc announced that the US FDA approved patisiran (Onpattro) infusion for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs.

It is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.

siRNAs work by silencing a portion of the RNA involved in causing the disease.

Patisiran encases the siRNA into a lipid nanoparticle to deliver the drug directly into the liver, in an infusion treatment, to alter or halt the production of disease-causing proteins.

Patisiran is designed to interfere with RNA production of an abnormal form of protein transthyretin (TTR). By preventing the production of TTR, the drug can help reduce the accumulation of amyloid deposits in peripheral nerves, improving symptoms and helping patients better manage the condition.