Achillion’s Danicopan (ACH-4471) Receives FDA’s Breakthrough Therapy Designation for Paroxysmal Nocturnal Hemoglobinuria

Shots:

• The FDA’s BT designation is based on safety & efficacy data of ongoing P-II study assessing Danicopan in combination with C5 mAb for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are suboptimal responders to C5 inhibitor monothx. with its expected results in Q4’19
•  The US FDA’s BT designation expedite the development and review of drugs for serious or life-threatening conditions
• The Danicopan is an orally active Factor D inhibitor, act by limiting both intravascular and extravascular hemolysis and has received FDA’s ODD in 2017 with its expected onset of P-III study in H1’2020

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