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Treatment Products

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Treatment Products

for Hemophilia

1. Factor Replacement Therapies

Recombinant Factor VIII (rFVIII)

For Hemophilia A

Synthetic factor VIII made using recombinant DNA technology. First-line for Hemophilia A. Brands include Advate, Kogenate, Eloctate (extended half-life).

Recombinant Factor IX (rFIX)

For Hemophilia B

Synthetic factor IX product. Preferred in Hemophilia B. Extended half-life versions (e.g., Alprolix, Idelvion) reduce infusion frequency.

Plasma-Derived Factor VIII or IX

Alternative for Hemophilia A or B

Made from donated human plasma. May carry a small risk of viral transmission. Includes von Willebrand factor in some formulations.

2. Non-Factor Replacement Therapy

Emicizumab (Hemlibra)

For Hemophilia A (with or without inhibitors)

A bispecific monoclonal antibody that mimics factor VIII function. Administered subcutaneously, typically weekly or every 2–4 weeks. Reduces bleeding episodes significantly.

3. Bypassing Agents (for Inhibitor Patients)

Recombinant Activated Factor VII (rFVIIa)

For Hemophilia A or B with inhibitors

Promotes clot formation independently of factor VIII or IX. Used for acute bleeding or surgery. Brand: NovoSeven.

Activated Prothrombin Complex Concentrate (aPCC)

For Hemophilia A or B with inhibitors

Contains activated clotting factors that bypass the need for factor VIII/IX. Used in on-demand or prophylactic regimens. Brand: FEIBA.

4. Adjunctive Therapies

Desmopressin (DDAVP)

For mild Hemophilia A

Stimulates release of stored factor VIII and von Willebrand factor. Administered IV or intranasally. Not useful for Hemophilia B or severe cases.

Antifibrinolytics (Tranexamic Acid, Aminocaproic Acid)

Supportive

Prevents breakdown of clots. Useful in mucosal bleeding, dental procedures, or menorrhagia. Often used alongside other therapies.

5. Gene Therapy (Emerging)

Etranacogene dezaparvovec (Hemgenix)

For Hemophilia B

FDA-approved gene therapy delivering a functional copy of the factor IX gene via an AAV vector. One-time IV infusion. May reduce/eliminate need for further factor therapy.

Valoctocogene roxaparvovec (under study)

For Hemophilia A

Experimental gene therapy using an AAV5 vector to deliver factor VIII gene. Demonstrates long-term FVIII expression in some patients.

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