RNA Targeting Drug Wins Approval for Rare Genetic Disorder

Akcea Therapeutics won FDA approval for Tegsedi (inotersen) — the first RNA-targeting drug of its kind — for treatment of peripheral nerve damage in patients with the rare genetic disorder, hereditary transthyretin-mediated amyloidosis.

The disease, in which proteins called transthyretin (TTR) proteins misfold and accumulate as amyloids throughout the body, is so rare that the government doesn’t have statistics on its frequency. It can be fatal.

Akcea, an affiliate of Ionis Pharmaceuticals, says that patients injected with Tegsedi in a four-year Phase II/III trial showed a 79 percent decrease in the amount of TTR proteins in their bodies.