Indications and Limitations of Use: ADYNOVATE® [Antihemophilic factor (Recombinant), PEGylated] is a human antihemophilic factor indicated for use in children and adults with hemophilia A for the control and prevention of bleeding episodes. ADYNOVATE is not indicated for the treatment of von Willebrand disease.1

Clinical experience—patient
reported outcomes

study design

In children under 12 years old: This prospective open-label, uncontrolled pivotal phase 3 study evaluated the efficacy, PK, and safety of 40–60 IU/kg ADYNOVATE twice-weekly prophylaxis and determined the ability to treat bleeding episodes for 6 months in 66 previously treated children under 12 years old with severe hemophilia A. The primary endpoint was to assess the development of FVIII inhibitors.1

In adolescents and adults 12 years and older: This open-label, nonrandomized, pivotal phase 2/3 study evaluated the efficacy, PK, and safety of ADYNOVATE twice-weekly prophylaxis vs on-demand treatment and determined hemostatic efficacy in the treatment of bleeding episodes for 6 months in 137 previously treated patients 12 years and older with severe hemophilia A. The primary study goal was to compare the ABR between the prophylaxis vs on-demand treatment regimens.1

SELECTED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS: Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
See Detailed Important Risk Information below.

study results

In 2 separate clinical studies, patients receiving ADYNOVATE reported:

Adolescents and adults 12 years and older:

Improvement in both bleed and pain severity was seen after 6 months of prophylaxis2

  • These results were considered clinically meaningful, but the mean changes from baseline were not statistically significant relative to changes reported in the on-demand group2
  • Prophylaxis patients (n=82) reported a mean improvement in bleed severity (-4.17), pain severity (-1.22), and the total symptom score (-2.70). On-demand patients (n=11) reported a mean improvement in bleed severity (-4.24), pain severity (-0.17), and in the total score (-2.20)2

Children under 12:

Overall, a reduction in pain and increase in physical activity were seen after 6 months of prophylaxis2

  • Overall, children under 12 (n=65) showed a reduction in pain (mean change from baseline: -11.82) as assessed by Visual Analog Pain Scale. When broken out by age, the results for patients <6 years were not significant2
  • Overall, children under 12 (n=65) showed an increase in physical activity (mean change from baseline: 13.05) as assessed by Physical Activity Question Set. When broken out by age, the results for patients 6–12 years were not significant2

Baseline 21.40

Baseline 72.12

SELECTED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS: Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
See Detailed Important Risk Information below.

Joint-bleed assessment

study design

In children under 12 years old: This prospective open-label, uncontrolled pivotal phase 3 study evaluated the efficacy, PK, and safety of 40–60 IU/kg ADYNOVATE twice-weekly prophylaxis and determined the ability to treat bleeding episodes for 6 months in 66 previously treated children under 12 years old with severe hemophilia A. The primary endpoint was to assess the development of FVIII inhibitors.3

SELECTED IMPORTANT RISK INFORMATION

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions: Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. 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.
See Detailed Important Risk Information below.

study results

In a clinical study of patients under 12 years old
(N=66), 14 patients had target joints§ at screening3

— 10 of 14 patients had at least 1 target joint revert to a non-target joint

of these patients (8 of 10),
all target joints resolved3

§A target joint is defined as a single joint (ankles, knees, hips, or elbows) with ≥3 spontaneous bleeding episodes in any consecutive 6-month period. Target joint resolution is defined as zero bleeds into a target joint over a consecutive 6-month period.

SELECTED IMPORTANT RISK INFORMATION

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions: Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. 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.
See Detailed Important Risk Information below.

Real-world experience with
patient adherence4

study design

A retrospective, observational, U.S.-based analysis was conducted from November 2015 to September 2017 to describe the clinical profiles of 56 patients with hemophilia A, between the ages of 5 and 88, who transitioned from prophylaxis with any rFVIII to ADYNOVATE prophylaxis. Some of the measured endpoints before and after switching from a previous rFVIII to ADYNOVATE included ABR (calculated for patients receiving ADYNOVATE greater than or equal to 3 months) and qualitative adherence. 4

Limitations
The limitations of this retrospective, observational study include: Patients were not randomized, observers were not blinded, and some subgroups (eg, patients <12 years of age, patients treated with a prior EHL rFVIII) were small in sample size. In addition, bleeding rates were patient reported. 4

Since these data are from a retrospective, observational chart review, they have less evidentiary value than prospective, controlled studies. Further confirmatory studies are required to draw any conclusions from these data.

See the safety data for ADYNOVATE

SELECTED IMPORTANT RISK INFORMATION

ADVERSE REACTIONS: The most common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
See Detailed Important Risk Information below.

study results

In a retrospective, observational, real-world study of ADYNOVATE patients:

reported good to complete adherence4

The definition of adherence was considered according to the percentage of prescribed doses that were taken: 100% = complete adherence; 80%—99% = good adherence; 50%—79% = moderate adherence; <50% =poor adherence4

Data from the real-world study on overall ADYNOVATE adherence were provided for 56 patients:4

  • 80% (45/56) of patients indicated good to complete adherence
  • 13% (7/56) of patients indicated moderate adherence
  • 5% (3/56) of patients indicated poor adherence
  • 1 patient did not specify their adherence to ADYNOVATE

For patients less than 12 years of age, 91% (10/11) indicated good to complete adherence to ADYNOVATE4

For patients 12 years and older, 78% (35/45) indicated good to complete adherence to ADYNOVATE4

SELECTED IMPORTANT RISK INFORMATION

ADVERSE REACTIONS: The most common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
See Detailed Important Risk Information below.

Meet Aaron

Diagnosed with hemophilia before being born, Aaron didn’t get his first bleed until he was nine months old. From then on, his home care nurse infused him on-demand. Then Aaron’s parents starting taking over the infusions when he was two. He says, “My parents say that I never ran or hid from my infusion, and they tell me they even remember me watching the procedure.” Aaron himself, learned to infuse at hemophilia camp, which was a family tradition every summer. It was a great place for him to meet other boys his age and learn about his condition.

Today, Aaron treats his hemophilia A prophylactically with ADYNOVATE. He became interested in the treatment after hearing about the extended half-life. Aaron admits that he sometimes felt like prophylaxis treatment was a chore. So, having a simple twice weekly dosing schedule on the same two days each week was important to him.

SELECTED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS: Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
See Detailed Important Risk Information below.

Proven bleed control

The efficacy of ADYNOVATE as a prophylaxis was studied in clinical trials

Get the results
Helping with on-demand needs

See ADYNOVATE bleed control data in children and adults from an on-demand clinical trial

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