The US Food and Drug Administration (FDA) has approved a factor X product derived from human plasma (Coagadex) to treat patients with hereditary factor X deficiency who are 12 years of age and older.
Coagadex is approved for on-demand treatment and control of bleeding episodes in these patients as well as for perioperative management of bleeding in patients with mild hereditary factor X deficiency.
Prior to this approval, there was no specific coagulation factor replacement therapy available for patients with hereditary factor X deficiency in the US. The FDA previously granted Coagadex orphan product designation, fast track designation, and priority review.
The FDA based its approval of Coagadex on results from 2 phase 3 trials of patients age 12 and older.
The first trial included 16 patients who received Coagadex for pharmacokinetic evaluation, on-demand treatment and control of bleeding episodes, and/or perioperative management of minor surgical or dental procedures.
Coagadex was used to treat 208 bleeding episodes, and 187 of these episodes (in 15 patients) were evaluated for efficacy. Ninety-eight episodes were major bleeds, 88 were minor bleeds, and 1 was not assessed.
One hundred and fifty-five bleeds (83%) were treated with a single infusion of Coagadex, 28 (15%) were treated with 2 infusions, 3 bleeds (2%) required 3 infusions, and 1 bleed (0.5%) required 4 infusions. Four bleeding episodes in 2 patients were considered treatment failures.
The mean dose of Coagadex per infusion was 25.4 IU/kg, and the mean total dose was 30.4 IU/kg. The recommended dose of 25 IU/kg to treat a bleed was maintained for 14 of the 16 patients. The other 2 patients used doses of up to 30 IU/kg and 33 IU/kg.
There were 176 adverse events in this trial, but only 6 events in 2 patients were considered possibly related to Coagadex. This included 2 reports of infusion site erythema in 1 patient, 2 reports of fatigue in 1 patient, 1 report of back pain, and 1 report of infusion site pain.
The second trial included patients who received Coagadex for perioperative management. Five patients received Coagadex for 7 surgical procedures.
For major surgeries, a median of 13 infusions (range, 2-15) and a median cumulative dose of 181 IU/kg (range, 45-210 IU/kg) were required to maintain hemostasis. For minor surgeries, a median of 2.5 infusions (range, 1-4) and a median cumulative dose of 89 IU/kg (range, 51-127 IU/kg) were required to maintain hemostasis.
There were no adverse events related to Coagadex in this trial.
For more details on these trials, see the Coagadex package insert. Coagadex is manufactured by Bio Products Laboratory Limited in Elstree, Hertfordshire, UK.
