Overview
Moroctocog alfa, also known as BDDrFVIII (B domain deleted recombinant factor VIII), is a recombinant DNA-based drug with functional characteristics comparable to those of endogenous coagulation Factor VIII, the essential human blood clotting protein that is impaired in Hemophilia A. Moroctocog alfa is identical in sequence to endogenously produced Factor VIII, but does not contain the B-domain, which has no known biological function. Moroctocog alfa is produced through recombinant DNA technology and purification, resulting in a 1438 amino acid, 170 kDa protein . Clinical evaluation has shown that BDDrFVIII is pharmacokinetically equivalent to full-length recombinant FVIII . Also known as Anti-Hemophilic Factor (AHF), endogenous Factor VIII is essential to the clotting process in the body due to its involvement in the clotting cascade where it is responsible for acting as a co-factor to Factor IX. Activation of Factor IX leads to a cascade of signals that results in activation of Factor X, which then results in the conversion of prothrombin to thrombin, and as a result, leads to the conversion of fibrinogen to fibrin, the fibrous protein that creates the scaffold of the clot. Replacement of Factor VIII is essential for the treatment of Hemophilia A, which is caused by mutations in the Factor VIII gene, leading to a functional deficiency or complete loss of protein. Congenital loss or deficiency of Factor VIII results in the physiologic impairment of the coagulation clotting cascade, and as a result, leads to easy bruising and bleeding. Bleeding can range in severity from minor concerns, such as nosebleeds, to more serious events such as hemorrhaging in the joints, brain, or digestive tract . Exogenous replacement of Factor VIII is currently the cornerstone of Hemophilia treatment and is used for the prophylaxis and control of bleeding episodes. Treatment has drastically improved since the 1960s when Factor VIII protein was primarily purified from human plasma, rather than being produced through recombinant DNA technology. Unfortunately, purification of protein from human plasma carries an increased risk of transmission of blood-borne diseases such as HIV and Hepatitis, which in part contributed to the Tainted Blood Scandal in the 1980s . Use of recombinant DNA-derived clotting factor treatments, such as Moroctocog alfa, has reduced this risk. Other drug products with similar structure and function to Moroctocog alfa include Antihemophilic factor human, which is purified Factor VIII from human pooled blood and contains both A- and B-subunits, and Efmoroctocog alfa, which is a fully recombinant factor VIII-Fc fusion protein which has an extended half-life compared with conventional factor VIII due to conjugation to the dimeric Fc domain of human immunoglobulin G1, a long-lived plasma protein . Moroctocog alfa is approved by Health Canada and by the European Medicines Agency for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia). As it does not contain von Willebrand factor it is not indicated in von Willebrand’s disease .
Indication
Moroctocog alfa is indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for on-demand treatment and control of bleeding episodes, perioperative management, and routine prophylaxis to reduce the frequency of bleeding episodes.
Associated Conditions
- Bleeding caused by Hemophilia A
Research Report
Moroctocog Alfa (BDDrFVIII): A Comprehensive Monograph on its Molecular Design, Clinical Efficacy, and Therapeutic Role in Hemophilia A
Executive Summary
Moroctocog alfa is a high-purity, B-domain deleted recombinant coagulation Factor VIII (BDDrFVIII) that serves as a cornerstone therapy in the management of hemophilia A. This biotech therapeutic is engineered to replace the deficient or absent endogenous Factor VIII, thereby restoring the integrity of the intrinsic coagulation cascade and enabling effective hemostasis. Its approved indications are comprehensive, encompassing on-demand treatment of acute bleeding episodes, perioperative management for surgical procedures, and routine prophylaxis to prevent bleeds in patients of all ages, from newborns to adults.[1]
The development of Moroctocog alfa reflects the broader safety-driven evolution of biologic medicines. Marketed under trade names such as ReFacto, ReFacto AF, and Xyntha, its manufacturing process has been progressively refined. The transition to an albumin-free cell culture (AF-CC) process, coupled with advanced purification techniques that eliminate animal- and human-derived components, has established Moroctocog alfa as a third-generation recombinant Factor VIII product with an enhanced safety profile regarding pathogen transmission.[4]
Clinical evidence robustly supports its efficacy. Prophylactic regimens have been shown to significantly reduce bleeding frequency, with studies demonstrating low median annualized bleed rates (ABR) and a substantial proportion of patients remaining bleed-free.[6] In on-demand settings, the vast majority of bleeding events are controlled effectively with one or two infusions.[6] Pharmacokinetic studies have confirmed its bioequivalence to earlier formulations and, critically, its therapeutic equivalence to full-length recombinant Factor VIII products, validating its unique molecular design.[5]
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
---|---|---|---|---|---|
2019/08/19 | Phase 1 | UNKNOWN | |||
2013/02/13 | N/A | Completed | |||
2012/04/18 | Phase 1 | Completed | |||
2009/03/25 | Phase 3 | Completed | |||
2005/09/19 | N/A | Completed | Wyeth is now a wholly owned subsidiary of Pfizer |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
---|---|---|---|---|---|
No FDA approvals found for this drug. |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
---|---|---|---|
Authorised | 4/13/1999 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
Xyntha Powder and Solvent for Solution for Injection 500iu | SIN13792P | INJECTION, POWDER, FOR SOLUTION | 500IU/vial | 4/28/2010 | |
Xyntha Powder and Solvent for Solution for Injection 1000iu | SIN13794P | INJECTION, POWDER, FOR SOLUTION | 1000IU/vial | 4/28/2010 | |
Xyntha Powder and Solvent for Solution for Injection 250iu | SIN13791P | INJECTION, POWDER, FOR SOLUTION | 250IU/vial | 4/28/2010 | |
Xyntha Powder and Solvent for Solution for Injection 2000iu | SIN13793P | INJECTION, POWDER, FOR SOLUTION | 2000IU/vial | 4/28/2010 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
---|---|---|---|---|---|
No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
---|---|---|---|---|---|
XYNTHA POWDER AND SOLVENT FOR SOLUTION FOR INJECTION 500IU | N/A | N/A | N/A | 9/25/2020 |
TGA Drug Approvals
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
---|---|---|---|---|---|
XYNTHA SOLOFUSE | 02374056 | Powder For Solution - Intravenous | 250 UNIT / SYR | N/A | |
XYNTHA | 02309491 | Kit
,
Powder For Solution - Intravenous | 500 UNIT / VIAL | 1/23/2009 | |
XYNTHA SOLOFUSE | 02374080 | Powder For Solution - Intravenous | 2000 UNIT / SYR | 5/8/2012 | |
REFACTO | wyeth canada | 02245951 | Powder For Solution - Intravenous | 1000 UNIT / VIAL | 2/11/2003 |
XYNTHA | 02309483 | Powder For Solution
,
Kit - Intravenous | 250 UNIT / VIAL | 1/23/2009 | |
XYNTHA | 02309505 | Kit
,
Powder For Solution - Intravenous | 1000 UNIT / VIAL | 1/23/2009 | |
XYNTHA SOLOFUSE | 02374099 | Powder For Solution - Intravenous | 3000 UNIT / SYR | 5/8/2012 | |
XYNTHA SOLOFUSE | 02374064 | Powder For Solution - Intravenous | 500 UNIT / SYR | 9/27/2019 | |
XYNTHA | 02309513 | Powder For Solution
,
Kit - Intravenous | 2000 UNIT / VIAL | 1/23/2009 | |
XYNTHA SOLOFUSE | 02374072 | Powder For Solution - Intravenous | 1000 UNIT / SYR | 4/13/2012 |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
---|---|---|---|---|---|
REFACTO AF 1000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE EN JERINGA PRECARGADA | 99103007 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized | |
REFACTO AF 2000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE EN JERINGA PRECARGADA | 99103008 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized | |
REFACTO AF 500 UI, POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 99103002 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Not Commercialized | |
REFACTO AF 250 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE EN JERINGA PRECARGADA | 99103009 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized | |
REFACTO AF 250 UI, POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 99103001 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Not Commercialized | |
REFACTO AF 1000 UI, POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 99103003 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Not Commercialized | |
REFACTO AF 3000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE EN JERINGA PRECARGADA | 99103005 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized | |
REFACTO AF 2000 UI, POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 99103004 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Not Commercialized | |
REFACTO AF 500 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE EN JERINGA PRECARGADA | 99103006 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE EN JERINGA PRECARGADA | Uso Hospitalario | Commercialized |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Philippines FDA approvals found for this drug. |
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Saudi SFDA approvals found for this drug. |
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Malaysia NPRA approvals found for this drug. |
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
---|---|---|---|---|---|
No UK EMC drug information found for this drug. |
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