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Achillion's blood disorder drug gets US FDA's breakthrough therapy tag

The U.S. Food and Drug Administration granted the breakthrough therapy designation to Achillion Pharmaceuticals Inc.'s danicopan to treat a rare blood disorder.

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The Blue Bell, P.a.-based biopharmaceutical company said danicopan, or ACH-4471, was granted the status for use in combination with a drug that inhibits the C5 protein to treat patients with paroxysmal nocturnal hemoglobinuria, or PNH, who have partially responded to existing therapy such as Alexion Pharmaceuticals Inc.'s Soliris and Ultomiris.

PNH is a rare acquired, life-threatening disease characterized by the destruction of red blood cells, blood clots and impaired bone marrow function.

Achillion said the FDA's decision was backed by positive safety and efficacy data from an ongoing phase 2 trial of danicopan, results of which are expected in the fourth quarter. Danicopan had received the FDA's orphan drug designation in 2017.

Achillion President and CEO Joe Truitt said the company plans to work with the FDA to advance the development of danicopan into phase 3 studies in early 2020.