FDA Approves Alnylam's Rare Disease Drug

(RTTNews) - The U.S. Food and Drug Administration Friday approved Onpattro, Alnylam Pharmaceuticals Inc.'s (ALNY) rare disease drug. The approval makes it the first approval of a therapy that employs the RNA interference (RNAi) gene-silencing technique.

Onpattro is also 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.

"This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms. In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness and loss of mobility," said FDA Commissioner Scott Gottlieb, M.D.

Affecting about 50,000 people worldwide, hATTR is a rare condition. It is characterized by the buildup of abnormal deposits of protein fibers called amyloid in the body's organs and tissues, interfering with their normal functioning.

Onpattro is developed by Alnylam Pharmaceuticals, using Arbutus Biopharma's Lipid Nanoparticle (LNP) delivery technology. Credit Suisse analyst Alethia Young expects Onpattro sales to peak by 2023 at $1.5 billion.