Factor in the bloodstream rises and falls in patients treated prophylactically with factor. It is highest right after an infusion and lowest right before an infusion. The low-level right before infusion is called the trough level. Many factor products aim to keep trough levels at 1%. In a study of adults and adolescents who were treated with Esperoct® for prophylaxis, researchers found that extended half-life Esperoct® keeps trough levels in adults at or above 3% (moderate hemophilia) all of the time, and 5% (mild hemophilia) approximately 90% of the time, based on PK modeling.a,b,c
aTrough level goal is 1% for prophylaxis.
bData shown are from a study where 175 previously treated adolescents and adults received routine prophylaxis with Esperoct® 50 IU/kg every 4 days for 76 weeks. Pre-dose factor activity (trough) levels were evaluated at follow-up visits. Mean trough levels for adolescents (12-<18 years) were 2.7 IU/dL.
cSteady-state factor VIII (FVIII) activity levels were estimated in 143 adults and adolescents using PK modeling.
Esperoct® is an extended half-life (EHL) factor. In a study of adults and adolescents treated with Esperoct® for prophylaxis, researchers confirmed that the recombinant factor stays in the body longer than standard half-life products.d And that typically means dosing less often. This EHL, in combination with the ability of Esperoct® to keep factor levels in adults at or above 3% for 100% of the timee, leads to effective bleed protection.
dData shown are from a comparison study of 26 previously treated patients (PTPs) 18 years or older who received a 25, 50, or 75 IU/kg dose of their previous SHL product followed by the same dose of Esperoct®. To allow for comparison, all results were adjusted to a 50 IU/kg dose of each product.
eData shown are from a study where 175 previously treated adolescents and adults received routine prophylaxis with Esperoct® 50 IU/kg every 4 days for 76 weeks. Pre-dose factor activity (trough) levels were evaluated at follow-up visits. Mean trough levels for adolescents (12-<18 years) were 2.7 IU/dL.
The number of doses and length of treatment varies by both procedure and patient. Your hematologist and surgeon will work together to create the best treatment plan for your circumstances.
Measuring a factor’s half-life gives doctors, patients and caregivers the opportunity to compare the factor activity level of different products by calculating the time it takes the body to metabolize or inactivate half the amount of factor.
How long factor stays in the blood, however, is only half the battle. The other consideration is the factor level. The extended half-life of Esperoct® (22 hours on average in adults) offers higher factor levels for longer compared to standard half-life products.f
fData shown are from 42 adults who received a pharmacokinetic (PK) assessment around the first Esperoct® 50 IU/kg dose.
PEGylation is actually a well-established technology that has been used in other treatments for many conditions including hemophilia.
Let’s look at the numbers of the Pathfinder clinical program, research that evaluated Esperoct®:
- In 270 previously treated people with severe hemophilia A
- During five, multi-center clinical trials
- Over 80,425 exposure days (corresponding to 889 patient years of treatment)
Beyond the numbers, the sheer breadth of the Pathfinder trial represents the Novo Nordisk dedication to your safety, as well as the effectiveness of Esperoct®.
An “exposure day” (ED) is typically considered a unit of time (1 day) during which a patient receives replacement treatment. Considering EDs during a clinical trial is helpful since inhibitors typically develop in those with severe hemophilia A after a median treatment time of 10-15 days. After 50-75 EDs, the cumulative incidence of inhibitors plateaus. However, EDs don’t tell the whole story. Other considerations that affect safety considerations include the amount of factor, frequency of infusions, the type of factor concentrate, and more.
Esperoct® is available in five different dose strengths and each glass vial has a different color cap to help with identification:
Don’t attempt to infuse yourself until you have gone through infusion training. Ask your healthcare provider or hemophilia treatment center how you can receive reconstitution and infusion training. Some specialty pharmacies also offer training.
As a prophylaxis treatment, your healthcare provider is able to determine dosing based on a simple, standard calculation of 50 IU per kilogram (50 IU/kg) of weight for adults and adolescents, and 65 IU/kg for children younger than 12. Your physician may adjust to less or more frequent dosing to achieve a specific target level or based on bleeding episodes.
No, you cannot reuse your MixPro® syringe. Each vial of Esperoct® is accompanied by its own pre-filled syringe and contains enough 0.9% saline solution to reconstitute a single vial of factor.
Good question! Two minutes is the length of time it takes for the infusion itself and doesn’t include set-up before infusion or clean-up after infusion.
Your best first stop is your healthcare provider who can answer this question as someone who understands you, your hemophilia care plan, and the facilities available in your area. You can often find infusion training at your hemophilia treatment center (HTC), specialty pharmacies, or from your physician.
Do not attempt to do an infusion yourself unless you have been taught how by your healthcare provider or hemophilia treatment center Please see Prescribing Information and Instructions for Use for more information on administration.
Your regional Hemophilia Community Liaison (HCL) is the bridge between you and the greater Novo Nordisk hemophilia team. Each HCL helps plan and then attends educational meetings and community bleeding disorders events. They also attend national bleeding disorder conferences and provide education and information about the hemophilia products and services offered by Novo Nordisk. They work with the bleeding disorder community to offer support and understanding about life with hemophilia and share those insights with the greater Novo Nordisk hemophilia team.
NovoCare® is a support program created to help you along your journey. NovoCare® provides assistance with coordinating insurance coverage, paying for treatment, and more. From verifying coverage to re-authorizing your prescription, we may be able to help.
PAP provides free medication to eligible applicants. Should you qualify under the PAP guidelines, the prescribed dose of Esperoct® will be shipped to your home.g There is no registration charge or monthly fee for participating. If you’d like to see if you qualify, download and fill out the PAP application form.h
gProduct limits vary.
hThe Novo Nordisk Patient Assistance Program (PAP) is administered by NovoSecure™. To qualify for the PAP, patients must demonstrate financial need and must have attempted to find alternative reimbursement. Several factors are considered in evaluating financial need, including cost of living, size of household, and burden of total medical expenses. If the applicant qualifies under the PAP guidelines, a limited supply of the requested medication(s) will be shipped to the patient. Patients who qualify for PAP may be eligible to receive the prescribed Novo Nordisk product, for up to 1 year from the approval date. Product limits vary.
Nobody needs to walk this path alone. That’s why we offer the support of regional Hemophilia Community Liaisons to support you on your Esperoct® journey. Those with hemophilia A and their families can also find a wealth of support and understanding from the National Hemophilia Foundation (NHF), the Hemophilia Federation of America (HFA), and other advocacy groups.
Advocacy groups like the NHF and the HFA help bring the bleeding disorders community together. Their goals usually include amplifying the patient or caregiver voice, affecting public policy, fundraising, and protecting the interests of BD community.