On October 20, 2015, FDA approved Coagadex, which is derived from human plasma, for hereditary Factor X deficiency. Until this orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

Indication: For individuals aged 12 and older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, and for perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management of bleeding in patients with mild hereditary Factor X deficiency.

Adverse Events: The most common adverse drug reactions (frequency ≥ 5% of subjects) observed in clinical trials were infusion site erythema, infusion site pain, fatigue and back pain.

Citation: FDA approves first Factor X concentrate to treat patients with rare hereditary bleeding disorder. [news release]. Rockville, MD: United States Food and Drug Administration; October 20, 2015. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm468038.htm. Accessed October 23, 2015.