Akcea Therapeutics genetic disease treatment gets FDA approval

Akcea's once-weekly injection, Tegsedi, treats hereditary ATTR amyloidosis patients with polyneuropathy.

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Akcea Therapeutics Inc said on Friday its treatment, developed along with Ionis Pharmaceuticals , which targets a rare genetic disease was approved by the U.S. Food and Drug Administration.

Akcea’s once-weekly injection, Tegsedi, treats hereditary ATTR amyloidosis patients with polyneuropathy.

Polyneuropathy, a symptom of the disease, is the simultaneous malfunction of peripheral nerves in the body resulting in tingling, numbness and kidney dysfunction.

Tegsedi belongs to a class of drugs that uses the Nobel-prize winning RNA interference mechanism to manipulate ribonucleic acid, which interferes with or “silences” targeted genes and stops the formation of proteins that can cause diseases.

Tegsedi’s approval in the United States comes nearly three months after its approval in Europe.

Based on the U.S. approval of Tegsedi, Ionis will receive a $50 million milestone payment and both the companies will split the gains from the drug’s sale at a ratio of 60 to 40 in favor of Ionis, Akcea said.

Earlier this year, the drug regulator had declined to approve Akcea’s similar drug, Waylivra, which looked to treat a genetic disease causing fat accumulation in the blood.

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