WHAT COULD THE FUTURE HOLD?

See how researchers aim to tackle unspoken trade-offs and hemostasis In hemophilia A¹

Nikola lives with Hemophilia A

TREATING HEMOPHILIA A

The goal of all current and investigational hemophilia therapies, from FVIII replacement to nonfactor therapies, is to restore thrombin generation to achieve effective hemostasis.²

FVIII replacement, FVIII mimetics, and gene therapy assist in better clotting.^3,4 Natural anticoagulants such as TFPI, APC, PS, and AT play a role in inhibiting thrombin generation or function at various points in the coagulation cascade. Targeting these anticoagulant proteins with hemophilia therapies aims to promote hemostasis.^2,3

APC=activated protein C; AT=antithrombin; FVIII=Factor VIII; FX=Factor X; PS=protein S; TFPI=tissue factor pathway inhibitor

David lives with Hemophilia A

Hemophilia Treatment Strategies of Today

—and ONGOING RESEARCH FOR Tomorrow

Click the expanders below to learn more about current and investigational hemophilia treatment approaches and what they could mean for your patients.

FVIII REPLACEMENT

FVIII MIMETICS

ANTICOAGULATION INHIBITION

GENE THERAPY

FIX=Factor IX; FIXa= Factor IXa; FVIIIa=Activated Factor VIII

IS NEAR-NORMAL
HEMOSTASIS POSSIBLE?

CERTAIN FVIII REPLACEMENT CAN MAINTAIN

FVIII LEVELS >40% for most of the week, which is in the near-normal range while current FVIII mimetics provide an estimated FVIII level equivalence of approximately 10% to 20% based on modeling and indirect assays.^11,12

Reduced thrombin generation,

independent of FVIII activity level, has been associated with a more severe bleeding phenotype.¹³

Depending on the class of therapy,

thrombin generation may be one way to measure hemostatic success.^2,14

What role does thrombin generation play in achieving near-normal hemostasis?

DOSING FREQUENCY IS
IMPORTANT

IN HEMOPHILIA MANAGEMENT, INCLUDING WHEN ADHERING TO A DOSING REGIMEN, ACCORDING TO SOME PATIENTS.^15,16

could fewer infusions or injections help reduce their management challenges?

Researchers are exploring how to design molecules to evolve pharmacokinetics, such as half-life and clearance rates, which may impact dosing regimens.⁹

Bentley lives with hemophilia A (and mom Teilei)

STAY UP TO DATE
WITH THE LATEST INFORMATION

Get updates and communications on hemophilia A from Novo Nordisk as they become available.

References

Data on file. Novo Nordisk Inc; Plainsboro, NJ
Sidonio RF Jr, Hoffman M, Kenet G, Dargaud Y. Thrombin generation and implications for hemophilia therapies: a narrative review. Res Pract Thromb Haemost. 2022;7(1):100018.
Ellsworth P, Ma A. Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates?. Hematology Am Soc Hematol Educ Program. 2021;2021(1):219-225.
Frequently Asked Questions. NBDF. National Bleeding Disorders Foundation. Accessed February 4, 2025. https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies/frequently-asked-questions
Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition [published correction appears in Haemophilia. 2021;27(4):699]. Haemophilia. 2020;26(suppl 6):1-158.
Swan D, Mahlangu J, Thachil J. Non-factor therapies for bleeding disorders: a primer for the general haematologist. EJHaem. 2022;3(3):584-595.
Ozelo MC, Yamaguti-Hayakawa GG. Impact of novel hemophilia therapies around the world. Res Pract Thromb Haemost. 2022;6(3):e12695.
Current Treatments. NBDF. National Bleeding Disorders Foundation. Accessed September 8, 2024.https://www.bleeding.org/bleeding-disorders-a-z/treatment/current-treatments
Teranishi-Ikawa Y, Soeda T, Koga H, et al. A bispecific antibody NXT007 exerts a hemostatic activity in hemophilia A monkeys enough to keep a nonhemophilic state. J Thromb Haemost. 2024;22(2):430-440.
Lillicrap D, Lenting P. Hemophilia A treatment innovation: factor VIII mimetic bispecific antibodies-generational enhancement. J Thromb Haemost. 2024;22(2):352-355.
ALTUVIIIO^® Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA.
Mancuso ME, Croteau SE, Klamroth R. Benefits and risks of non-factor therapies: redefining haemophilia treatment goals in the era of new technologies. Haemophilia. 2024;30 Suppl 3:39-44.
Verhagen MJA, van Balen EC, Blijlevens NMA, et al. Patients with moderate hemophilia A and B with a severe bleeding phenotype have an increased burden of disease. J Thromb Haemost. 2024;22(1):152-162.
Bernardo Á, Caro A, Martínez-Carballeira D, et al. Applicability of the thrombin generation test to evaluate the hemostatic status of hemophilia A patients in daily clinical practice. J Clin Med. 2022;11(12):3345.
Tischer B, Marino R, Napolitano M. Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice. Patient Prefer Adherence. 2018;12:431-441.
Thornburg CD, Duncan NA. Treatment adherence in hemophilia. Patient Prefer Adherence. 2017;11:1677-1686.

See how researchers aim to tackle unspoken trade-offs and hemostasis In hemophilia A1

See how researchers aim to tackle unspoken trade-offs and hemostasis In hemophilia A¹