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
Bleed protection for children and adults with proven results
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 clinical studies with patients receiving twice-weekly prophylaxis, ADYNOVATE demonstrated:
zeroBleeding EpisodesIn 38% of children under 12 years old (25 out of 66)1In 40% of adolescents and adults 12 years and older (40 out of 101 in the per-protocol group)1Bleeds or Less per YearIn children and adults†1
†Data presented here are from 2 different trials and are based on median overall annualized bleed rate results.
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.
Striving toward a goal of ZERO bleeds with ADYNOVATE prophylaxis for children and adults1
During the 6-month prophylaxis clinical study in children under 12 (N=66)1,2:
had zero total bleeding episodes1
had zero spontaneous bleeding episodes1
had zero joint bleeding episodes1
During the 6-month prophylaxis study in adolescents and adults (12 years and older)1:
‡n=40/101, results from per-protocol group
had zero total bleeding episodes1
had zero spontaneous bleeding episodes3
had zero joint bleeding episodes1
In clinical studies with patients receiving twice-weekly prophylaxis, ADYNOVATE demonstrated:
Overall median abr
Prophylaxis
On-demand
In children <12Results from full analysis set
2.0 (IQR, 3.9) (n=66)1,3
N/A
In adolescents and adults ≥12
1.9 (IQR 5.9) (n=120)1,3
41.5 (IQR, 19.4) (n=17)1,3
Spontaneous median abr
Prophylaxis
On-demand
In children <12Results from full analysis set
0.0 (IQR, 1.9) (n=66)1,3
N/A
In adolescents and adults ≥12
0.0 (IQR 2.2) (n=120)1,3
21.6 (IQR, 22.0) (n=17)1,3
Joint median abr
Prophylaxis
On-demand
In children <12Results from full analysis set
0.0 (IQR, 1.9) (n=66)1,3
N/A
In adolescents and adults ≥12
0.0 (IQR 2.0) (n=120)1,3
38.1 (IQR, 20.1) (n=17)1,3
Median overall abr
spontaneous median abr
joint median abr
Prophylaxis
On-demand
Prophylaxis
On-demand
Prophylaxis
On-demand
In children <12Results from full analysis set
2.0 (IQR, 3.9) (n=66)1,3
N/A
0.0 (IQR, 1.9) (n=66)1,3
N/A
0.0 (IQR, 1.9) (n=66)1,3
N/A
In adolescents and adults ≥12
1.9 (IQR 5.9) (n=120)1,3
41.5 (IQR, 19.4) (n=17)1,3
0.0 (IQR 2.2) (n=120)1,3
21.6 (IQR, 22.0) (n=17)1,3
0.0 (IQR 2.0) (n=120)1,3
38.1 (IQR, 20.1) (n=17)1,3
ABR=annualized bleeding rate; IQR=interquartile range
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 study results
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.
Neutralizing Antibodies: Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.
See Detailed Important Risk Information below.
study results
In a retrospective, observational, real world study, it was reported that ADYNOVATE has shown:
median ABR for overall study population4
In the retrospective, observational, real-world study, the median ABR was 0.8 for those who received prior SHL rFVIII (n=35) and for those who received a prior EHL rFVIII (n=3), the median ABR was 2.44
For patients 12 and over, the median ABR was 0.8 and the mean ABR was 1.7 (n=33)4
of patients (20 out of 51) reported ZERO bleeding episodes4
SELECTED IMPORTANT RISK INFORMATION
WARNINGS & PRECAUTIONS
Neutralizing Antibodies: Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.
See Detailed Important Risk Information below.
Meet Dylan
Hemophilia A has been part of Dylan’s life since he was 8 months old. Initially, he was treated on-demand at the clinic or the emergency room when he had a bleed. As he got a little older, Dylan began seeing a hemophilia specialist, who talked to his mother about treating prophylactically. Since Dylan was experiencing a lot of joint bleeds, prophylaxis made a lot of sense, and he began a regimen soon after.
Dylan admits living with and treating his condition hasn’t always been easy, but he’s learned a few things along way. Like how to self-infuse. "When I was 11 years old I had the opportunity to travel to Canada as a student ambassador, and my mom told me that unless I could self-infuse my factor, I wouldn’t be allowed to go." Dylan’s mom arranged for a nurse to come by the house two or three times a week to teach him how to infuse until he got comfortable with the process. And Dylan ended up learning how, and going on his trip.
Today, Dylan treats with ADYNOVATE. He and his mother had a conversation with his doctor about the risks and benefits of ADYNOVATE and all decided it was right for him. Since being on ADYNOVATE, Dylan's bleeding has been under control.
Dylan: actual patient
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.
Helping with on-demand needs
See ADYNOVATE bleed control data in children and adults from an on-demand clinical trial
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‡n=40/101, results from per-protocol group
