Coagadex now available for rare bleeding disorder patients in the UK

Treatment for rare bleeding disorder ‘hereditary factor X deficiency’ is made available in the UK

Bio Products Laboratory, Limited (BPL) today announced that Coagadex is now available for patients in the UK. Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency. Coagadex is the only treatment licensed specifically for this rare bleeding disorder in Europe1, and the UK is now the first country in Europe to make this treatment available to patients.

Coagadex will be made available in the UK via a Commercial Medicines Unit (CMU) tender, which starts on July 1^st. The CMU works on behalf of the Department of Health and the NHS, and in partnership with the people who buy pharmaceuticals for hospitals across the NHS in the UK.

Factor X deficiency is a rare and serious condition caused by not having enough of the factor X protein in the blood; this protein plays a crucial role in coagulation (blood clotting) which helps patients stop bleeding. People with factor X deficiency are at increased risk of bleeding or experiencing excessive or prolonged bleeding. Severely affected individuals have an increased risk of bleeding inside the brain, in the lungs or in the gastrointestinal tract, which can be life-threatening. Hereditary factor X deficiency is very rare and affects approximately 700 patients in Europe.

Dr Steve Austin, Haemophilia Centre Director of St George’s University Hospitals NHS Foundation Trust, London commented:

Our previous focus has been on treating Factor X deficiency with blood infusions of plasma or a concentrate of clotting factors. Having been involved in the clinical development programme of Coagadex, I am delighted that this therapy has now been launched in the UK. For the first time, patients with this rare bleeding disorder can receive a specific factor X replacement that has been proven safe and effective in clinical studies.

John Perkins, CEO of BPL, commented:

Coagadex is the first and only specifically approved therapy to treat hereditary factor X deficiency and is now available to the patients who need it in the UK. This achievement is a demonstration of our commitment to patients in areas with the greatest unmet need.

Coagadex was approved based upon data generated from two open-label, multicentre, prospective studies. The first study enrolled patients with moderate to severe hereditary factor X deficiency who were treated on-demand for spontaneous or traumatic bleeding episodes. The primary efficacy endpoints were pharmacokinetic measures including recovery rate and half-life, and secondary endpoints included overall assessment of efficacy and the number of infusions needed to treat a bleed.

The criteria for treatment success were satisfied in the study, and the pharmacokinetic parameters were consistent with previously published data. The overall mean in-vivo recovery rate was 2.0 IU/dL per IU/kg and the half-life was approximately 30 hours. There were 187 assessable bleeds in the study with patients rating the treatment as "excellent" in 170 (91%) cases, "good" in 14 (7.5%) cases, and "poor" in 2 (1.1%) cases. In addition, most bleeding episodes (155/187 [82.9%]) were effectively treated with only one infusion of Coagadex.

Two patients in the study reported six adverse events considered possibly related to the medication: two events of fatigue in one patient, two events of infusion site erythema in one patient, and one of infusion site pain and back pain in each patient. There were no other drug-related adverse events, no serious drug-related adverse events, and no patients discontinued from the study due to adverse events.

The second study collected data on two surgical patients receiving Coagadex perioperatively. Surgical data from three patients in the first study was added and resulted in five patients undergoing seven surgical procedures. For all surgical procedures, Coagadex was assessed by the investigator as excellent in controlling blood loss during and after surgery. All patients undergoing major surgery were diagnosed with mild factor X deficiency (i.e., factor X level >5 IU/dL and < 20 IU/dL). One patient with moderate deficiency and two with severe deficiency underwent minor procedures. No patients with moderate or severe disease underwent a major procedure. There were no treatment-related adverse events reported in surgical patients in the second study.

In both studies combined, the most common adverse events were infusion site erythema, infusion site pain, fatigue and back pain.

The launch of Coagadex follows the recent marketing authorisation approval by the European Commission in March 2016.