On-demand Trial Results: Overall Efficacy

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Methods

COAGADEX was studied in a prospective, open-label, multicenter, non-randomized phase III study in 16 adult and adolescent subjects (≥12 years old) with severe/moderate factor X deficiency (Study TEN01)

  • COAGADEX was administered on-demand or for short-term prophylaxis for 6 months to 2 years until ≥12 bleeding episodes had been treated with the product
  • Key inclusion criteria:
    • Aged ≥12 years
    • Baseline plasma factor X concentration <5 IU/dL
    • Factor X replacement therapy for ≥1 spontaneous or menorrhagic bleeds in the past year
  • Key exclusion criteria:
    • History of factor X inhibitor development
    • Positive for factor X inhibitors at the screening visit
    • Known thrombocytopenia or clinically significant renal or liver disease

Results

In this study, 87.3% of non-menorrhagic bleeds were covert

  • In this study, 16 subjects experienced a total of 187 bleeds that were assessed by subjects and an independent data review committee consisting of 3 hemophilia experts
Types of Bleeds that Occurred in the Trial n (%) of bleeds N=187
Bleed type
Covert 110 (58.8)
Menorrhagic 61 (32.6)
Overt 16 (8.6)
Bleed location
Mucosal 73 (39.0)
Joint 63 (33.7)
Muscle 26 (13.9)
Other* 25 (13.4)
Bleed cause
Spontaneous 79 (42.2)
Menorrhagia 61 (32.6)
Injury 47 (25.1)
Bleed severity
Major 98 (52.4)
Minor 88 (47.1)
Not evaluated 1 (0.5)

*Includes cut/wound, concussion, foot, forehead, hand, kidney, left forearm, lumbar area, right shoulder, subcutaneous and thumb.

As categorized by the data review committee.

  • 98% of bleeds were controlled with 1 or 2 infusions of COAGADEX

    Infusions of COAGADEX Needed to Control Bleeding

    Infusions of COAGADEX Needed to Control Bleeding

    • Mean (SD) dose per infusion was 25.3 (2.4) IU/kg
  • 98.4% of bleeds were treated successfully (defined as “excellent” or “good response”)

    Subject Assessment of Treatment Response with COAGADEX

    Subject Assessment of Treatment Response with COAGADEX

Rating Scale for Efficacy of COAGADEX

Efficacy rating Overt bleeds Covert bleeds Menorrhagic bleeds
Excellent Bleeding stopped within 12h after dosing with COAGADEX, with only 1 dose required 1 dose of COAGADEX was required or 2 doses of COAGADEX were required less than 48h apart 1 dose of COAGADEX was required or 2 doses of COAGADEX were required less than 48h apart
Good Bleeding stopped within 24h after the first dose of COAGADEX, with 1 or 2 doses required 3 doses of COAGADEX were required, with less than 48h between the first and last dose 2 doses of COAGADEX were required, with more than 48h between the first and the last dose

Reference: Austin S, Kavakli K, Norton M, et al. Haemophilia. 2016;22:419-425.

Indications and Usage for COAGADEX

COAGADEX, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children with hereditary factor X deficiency for:

Limitation of Use

Perioperative management of bleeding in major surgery in patients with severe hereditary factor X deficiency has not been studied.

Contraindication for COAGADEX

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

Important Safety Information for COAGADEX

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

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, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) 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.

Please see complete Prescribing Information for COAGADEX.