Bio Products Laboratory Presents New Phase 3 Data Demonstrating Prophylactic Coagadex® (Coagulation Factor X, Human) Dosing Prevents or Reduces Bleeding Episodes in Children with Moderate to Severe Hereditary Factor X Deficiency

ELSTREE, England and DURHAM, N.C., July 12, 2017 /PRNewswire/ -- Bio Products Laboratory Limited (BPL), a leading manufacturer of plasma-derived protein therapies, presented this week new data from TEN02, the Phase 3, prospective study of Coagadex® (Coagulation Factor X, Human) for prophylaxis of bleeding episodes in children <12 years old with moderate to severe hereditary factor X deficiency. Coagadex is currently the only FDA-approved product to treat hereditary factor X deficiency in adults and children >12. These data were presented at the 2017 International Society on Thrombosis and Haemostasis Congress being held July 8-13, 2017 in Berlin, Germany.

"Due to the harmful long-term effects of repeated bleeding episodes, a prophylactic regimen of a single factor concentrate for the prevention of bleeds is a recognized best practice in treating bleeding disorders," said Dr. Michael Gattens, Consultant Paediatric Haematologist, Cambridge University Hospitals, UK and one of the investigators in the TEN02 clinical trial. "For the first time, we have data providing physicians with important evidence of the efficacy and safety of Coagadex as a prophylactic regimen for the reduction and prevention of bleeding episodes, as well as potential guidance on dosing for children with hereditary factor X deficiency."

The primary endpoint of the study was the prophylactic efficacy of Coagadex, which was rated as "excellent" by the investigators for all subjects in the per-protocol population. A dosing regimen of 40–50 IU/kg twice weekly was recommended in the study; dose and frequency were adjusted over the initial six weeks to maintain factor X concentration levels ≥5 IU/dL (with peak levels ≤120 IU/dL). Data from the study showed that Coagadex was effective in preventing bleeding episodes in subjects <12 years old with moderate to severe factor X deficiency. Consistent with previous findings in subjects ≥12 years old, Coagadex was well tolerated and safe among subjects <12 years old.

These new data expand on the established efficacy of Coagadex, which was approved in October 2015 for the treatment of adults and children (aged 12 years and over) with hereditary factor X deficiency for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in patients with mild hereditary factor X deficiency. Coagadex is the first and only FDA-approved product to treat hereditary factor X deficiency and is the only specific factor X concentrate available in the U.S.

Hereditary factor X deficiency is a rare bleeding disorder that affects approximately 300-600 patients in the U.S. Affected individuals often have inadequate amounts of circulating factor X, an important component of the coagulation system. Factor X deficient patients are at increased risk of bleeding and need to be managed similarly to hemophilia patients.

In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with Coagadex were infusion site erythema, infusion site pain, fatigue and back pain.

Please see the Important Safety Information below and www.coagadex.com for full prescribing information.

The poster from the TEN02 study was published in a special July issue of Research and Practice in Thrombosis and Haemostasis and can also be found on the 2017 International Society on Thrombosis and Haemostasis Congress website.

TEN02 (ClinicalTrials.gov NCT01721681) was an open-label, multicenter, nonrandomized, phase 3, prospective study conducted in children <12 years with a diagnosis of moderate or severe congenital factor X deficiency (basal plasma factor X activity <5 IU/dL at diagnosis) and either a history of severe bleeding or an F10 gene mutation causing a documented severe bleeding type.

In the study, subjects received 50 IU/kg of Coagadex at visit 1 (baseline), followed 72 hours later by a second dose (visit 2); at the investigator's discretion, visit 2 may have been conducted 48 hours after the first dose for subjects aged 0–5 years. Following visit 2, subjects returned to the study site for 3 additional visits (after 9–28, 29–42, and 50 treatment days/26 weeks; visits 3–5), during which time blood samples were collected to assess FX:C trough levels and vital signs and adverse events (AEs) were checked. A final bolus dose of 50 IU/kg was administered during visit 5 to assess postdose incremental recovery.

Routine prophylaxis

• Following visit 2, subjects used pdFX 40–50 IU/kg as routine prophylaxis every 72 ± 2 hours (or every 48 ± 2 hours for subjects aged 0–5 years at the investigator's discretion).
• A dosing regimen of 40–50 IU/kg twice weekly was recommended; dose and frequency were adjusted over the initial 6 weeks to maintain FX:C levels ≥5 IU/dL (with peak levels ≤120 IU/dL).
• Treatment was continued for at least 26 weeks, with a minimum of 50 treatment days.

 Treatment of bleeds

• A dose of 25 IU/kg was recommended to treat minor bleeds; 50 IU/kg was recommended for major bleeds, and treatment was to be repeated as often as necessary based on FX:C recovery levels and clinical need.

Adverse Events

• A total of 28 adverse events (AEs) were reported in 8 children, none of which was considered related to treatment. The majority of AEs (n=26; 92.9%) were mild in severity; the remaining 2 AEs were of moderate severity. No deaths or other serious AEs were reported.

Indications for Coagadex

Coagadex, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children (aged 12 years and above) with hereditary Factor X deficiency for:

• On-demand treatment and control of bleeding episodes
• Perioperative management of bleeding in patients with mild hereditary Factor X deficiency

Limitation of Use
Perioperative management of bleeding in major surgery in patients with moderate and severe hereditary Factor X deficiency has not been studied.

Important Safety Information for Coagadex

Coagadex is contraindicated in patients with known hypersensitivity to any of the components of the product.

Allergic type hypersensitivity reactions, including anaphylaxis, are possible with Coagadex. If symptoms occur, patients should discontinue use of the product immediately and contact their physician.

The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking Coagadex for the development of inhibitors by appropriate clinical observations and laboratory tests.

Coagadex is made from human plasma and may contain infectious agents, e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of Coagadex.

In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with Coagadex were infusion site erythema, infusion site pain, fatigue and back pain.

For complete Prescribing Information for Coagadex please see www.coagadex.com.

About Bio Products Laboratory, Ltd.

Bio Products laboratory, Limited (BPL) is a leading manufacturer of plasma-derived protein therapies with global headquarters in Elstree, England, US headquarters in Durham, NC, and a presence in more than 45 countries worldwide.  The company has over 60 years of experience developing and manufacturing plasma-derived therapies since being established as part of the Lister Institute in 1950, and currently markets a wide range of products, including coagulation factors, human immunoglobulins, and albumin.  BPL is committed to continued investment in research and development to maintain its key position as a reliable supplier of high-quality products to patients and healthcare providers worldwide.

Contact:

Melyssa Weible
Elixir Health Public Relations
Ph: +1 201-723-5805
Email: [email protected]

SOURCE Bio Products Laboratory Limited

Related Links

http://www.coagadex.com