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ELOCTATE™ [Antihemophilic Factor (Recombinant), Fc Fusion Protein]
Lyophilized Powder for Solution For Intravenous Injection.
Brief Summary of Full Prescribing Information.
1 INDICATIONS AND USAGE
ELOCTATE, Antihemophilic Factor (Recombinant), Fc Fusion Protein, is a recombinant
DNA derived, antihemophilic factor indicated in adults and children with Hemophilia A
(congenital Factor VIII deficiency) for:
• Control and prevention of bleeding episodes,
• Perioperative management (surgical prophylaxis),
• Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
ELOCTATE is not indicated for the treatment of von Willebrand disease.
4 CONTRAINDICATIONS
ELOCTATE is contraindicated in patients who have had life-threatening hypersensitivity
reactions to ELOCTATE, including anaphylaxis.
5 WARNINGS AND PRECAUTIONS
5.1 Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, are possible with ELOCTATE. Early signs
of hypersensitivity reactions that can progress to anaphylaxis may include angioedema,
chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue
administration and initiate appropriate treatment if hypersensitivity reactions occur.
5.2 Neutralizing Antibodies
Formation of neutralizing antibodies (inhibitors) to Factor VIII can occur following
administration of ELOCTATE. Monitor all patients for the development of Factor VIII
inhibitors by appropriate clinical observations and laboratory tests. If the plasma
Factor VIII level fails to increase as expected or if bleeding is not controlled after
ELOCTATE administration, suspect the presence of an inhibitor (neutralizing antibody).
[see Monitoring Laboratory Tests (5.3)]
5.3 Monitoring Laboratory Tests
• Monitor plasma Factor VIII activity by performing a validated test (e.g., one stage
clotting assay), to confirm that adequate Factor VIII levels have been achieved and
maintained. [see Dosage and Administration (2)]
• Monitor for the development of Factor VIII inhibitors. Perform a Bethesda inhibitor
assay if expected Factor VIII plasma levels are not attained, or if bleeding is not
controlled with the expected dose of ELOCTATE. Use Bethesda Units (BU) to report
inhibitor levels.
6 ADVERSE REACTIONS
Common adverse reactions (≥1% of subjects) reported in clinical trials were arthralgia
and malaise.
6.1 Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction
rates observed in the clinical trials of one drug cannot be directly compared to rates in
clinical trials of another drug and may not reflect the rates observed in practice.
In the multi-center, prospective, open-label, clinical trial of ELOCTATE, 164 adolescent
and adult, previously treated patients (PTPs, exposed to a Factor VIII containing product
for ≥150 exposure days) with severe Hemophilia A (<1% endogenous FVIII activity or
a genetic mutation consistent with severe Hemophilia A) received at least one dose
of ELOCTATE as part of either routine prophylaxis, on-demand treatment of bleeding
episodes or perioperative management. A total of 146 (89%) subjects were treated for at
least 26 weeks and 23 (14%) subjects were treated for at least 39 weeks.
Adverse reactions (ARs) (summarized in Table 3) were reported for nine (5.5%) subjects
treated with routine prophylaxis or episodic (on-demand) therapy.
Two subjects were withdrawn from study due to adverse reactions of rash and arthralgia.
In the study, no inhibitors were detected and no events of anaphylaxis were reported.
Table 3: Adverse Reactions Reported for ELOCTATE (N=164)
MedDRA System Organ Class
MedDRA Preferred Term
2 (1.2)
1 (0.6)
1 (0.6)
1 (0.6)
Nervous system disorders
Dizziness
Dysgeusia
Headache
1 (0.6)
1 (0.6)
1 (0.6)
Musculoskeletal disorders
Arthralgia
Joint swelling
Myalgia
2 (1.2)
1 (0.6)
1 (0.6)
Gastrointestinal disorders
Abdominal pain, lower
Abdominal pain, upper
1 (0.6)
1 (0.6)
8.1 Pregnancy
Pregnancy Category C
Animal reproductive studies have not been conducted with ELOCTATE. It is not known
whether or not ELOCTATE can cause fetal harm when administered to a pregnant woman
or can affect reproduction capacity. ELOCTATE should be given to a pregnant woman only
if clearly needed.
8.3 Nursing Mothers
It is not known whether or not ELOCTATE is excreted into human milk. Because many
drugs are excreted into human milk, caution should be exercised when ELOCTATE is
administered to a nursing woman.
8.4 Pediatric Use
Pharmacokinetic studies in children have demonstrated a shorter half-life and lower
recovery of Factor VIII compared to adults. Because clearance (based on per kg body
weight) has been shown to be significantly higher in the younger, pediatric population
(2 to 5 years of age), higher and/or more frequent dosing based on body weight may be
needed. [see Clinical Pharmacology (12.3)]
Safety and efficacy studies have been performed in 5