Recombinate (antihemophilic factor recombinant) for hemophilia
Last updated April 4, 2025, by Margarida Maia, PhD
Fact-checked by Joana Carvalho, PhD
What is Recombinate for hemophilia?
Recombinate (antihemophilic factor recombinant) was a recombinant (lab-made) clotting factor VIII (FVIII) concentrate approved to prevent and control bleeds, including those occurring during surgery, in children and adults with hemophilia A.
Administered via an intravenous (into-the-vein) injection, the therapy was developed by the Genetics Institute of Boston and licensed to Baxter, now part of Takeda Pharmaceuticals. In March 2025, Takeda announced its decision to discontinue Recombinate because of an evolution in the treatment landscape, with patients transitioning to newer therapies.
Therapy snapshot
| Brand name: | Recombinate |
| Chemical name: | Antihemophilic factor recombinant |
| Usage: | Prevention and treatment of bleeding episodes, including those occurring during surgery, in hemophilia A |
| Administration: | Intravenous injection |
How does Recombinate work?
Hemophilia A is caused by mutations leading to a missing or faulty FVIII, a clotting protein in the blood. Without FVIII, the blood cannot clot normally. As a result, people with hemophilia A may experience heavy or prolonged bleeding episodes occurring either spontaneously or following an injury or surgical trauma.
Recombinate contained a recombinant version of FVIII, which was similar in structure and worked in the same way as human FVIII. It could be used as a replacement therapy to temporarily make up for the missing or faulty FVIII, helping to prevent and control bleeding in hemophilia A patients.
The recombinant protein was made in Chinese hamster ovary cells genetically modified in the lab by introducing a gene enabling them to make the recombinant protein.
Who could take Recombinate?
Recombinate was approved by the U.S. Food and Drug Administration in December 1992 to prevent and control bleeding episodes in people with hemophilia A. It was the first recombinant FVIII concentrate to be approved in the country for that indication.
It was also approved to manage bleeding episodes before, during, and after minor or major surgical procedures.
The therapy was approved in Europe in June 1993, but is no longer commercially available.
Who could not take Recombinate?
Recombinate was contraindicated, or not recommended, for people who previously experienced life-threatening allergic reactions to:
- any ingredient in Recombinate, including calcium, histidine, human albumin, polyethylene glycol, polysorbate-80, and sodium
- bovine, mouse, or hamster proteins.
Recombinate was also not indicated to treat von Willebrand disease, another bleeding disorder.
How was Recombinate administered in hemophilia?
Recombinate was given as a slow infusion directly into the bloodstream, and could be self-administered after proper training by a doctor or hemophilia center.
The concentrate was available as an off-white to faint yellow powder supplied in single-use vials that came in five color-coded strengths:
- light blue contained 250 international units (IU) of FVIII
- pink contained 500 IU of FVIII
- green contained 1,000 IU of FVIII
- purple contained 1,500 IU of FVIII
- orange contained 2,000 IU of FVIII.
The vials were packaged with 5 mL of sterile water for injection and a Baxject II needleless transfer device that allowed for an easy reconstitution, or dilution, process. When mixed together, powder and diluent created a clear, colorless to faint yellow solution that was ready to infuse within three hours of reconstitution.
How much Recombinate was given depended on factors such as where the bleeding was located and how heavy it was, as well as on a patient’s body weight.
For on-demand treatment, and until bleeding was resolved, patients should have been given a dose of Recombinate to maintain FVIII activity levels at:
- 20%-40% of normal, with dosing every 12-24 hours for one to three days, in the case of a minor bleeding episode (e.g., muscle or oral bleeds)
- 30%-60% of normal, with dosing every 12-24 hours for three days or more, in the case of more extensive bleeding (e.g., extensive muscle bleeding or hematoma)
- 60%-100% of normal, with dosing every 8-24 hours, in the case of a life-threatening bleeding episode (e.g., head injury or throat bleeding).
For a minor surgical procedure, such as tooth extraction, FVIII activity levels should be maintained at 60%-80% of normal. A single dose of Recombinate plus an oral antifibrinolytic medication given within one hour should have been sufficient to maintain FVIII activity levels in that range and control bleeding in 70% of cases.
For a major surgical procedure, Recombinate should have been given before surgery, and then every 8-24 hours to maintain FVIII activity levels at 80%-100% of normal until healing was complete.
The infusions could be done at a clinic or at home at a rate of up to 5 mL per minute, based on a patient’s comfort level. Patients were advised not to attempt to inject Recombinate unless they had received proper training on how to do the infusions from a doctor or a hemophilia treatment center.
Recombinate in hemophilia clinical trials
The safety and efficacy of Recombinate were evaluated in two open-label clinical studies in people with hemophilia A, including those who had been previously treated with a plasma-derived FVIII product and those who had not.
Trial in previously treated patients
In a 48-month study, 69 people with moderate or severe hemophilia A who had been previously treated with plasma-derived FVIII received Recombinate for preventive (prophylaxis) or on-demand treatment of bleeding episodes.
Among 3,481 bleeding episodes, response to treatment was rated good or excellent in 3,195 (91.2%). Recombinate also was considered excellent at preventing excessive bleeding in a total of 24 minor and major surgical procedures in 13 patients.
Treatment did not result in the development of inhibitors (neutralizing antibodies against FVIII), which may have stopped Recombinate from working properly.
Previously untreated patients
The other study included 79 previously untreated people with severe hemophilia A who received Recombinate for prophylaxis or on-demand treatment of bleeding episodes.
Of 810 bleeds, most (92%) were managed with one or two infusions of Recombinate. In all but one out of the 10 surgical procedures, bleeding control was rated excellent. In the other procedure, the patient developed inhibitors and experienced excessive bleeding after the surgery.
In total, 23 patients developed inhibitors against FVIII during the study, indicating that Recombinate was about equally as likely as plasma-derived FVIII to trigger the development of inhibitors.
Common side effects of Recombinate
The most common side effects reported with Recombinate during clinical trials were:
- chills
- flushing
- rash
- nose bleeds (epistaxis).
Allergic reactions
Allergic or hypersensitivity reactions, including anaphylaxis (a severe, whole-body allergic reaction), might have occurred in patients treated with Recombinate. Symptoms of an allergic reaction might have included dizziness, itching, rash or urticaria (hives), swelling, tightness of the throat, shortness of breath, paleness, fever, nausea, paresthesia (burning or prickling sensations), low blood pressure, and loss of consciousness.
Because Recombinate contained trace amounts of bovine, mouse, and hamster proteins, patients treated with Recombinate might have experienced allergies to these nonhuman proteins. If symptoms developed, treatment should have been stopped and emergency medical attention sought.
Development of inhibitors
Patients on Recombinate might have developed neutralizing antibodies, or inhibitors, against FVIII, which could have prevented the therapy from working as intended. The risk of developing inhibitors was highest in the first 20 days of treatment. This was most common in patients who had not been previously treated (or had been minimally treated) with replacement therapy.
If the expected FVIII activity levels were not reached, or if bleeding was not brought under control with an appropriate dose, a blood test might have been done to check for the presence of inhibitors.
Use in pregnancy and breastfeeding
It was not known if Recombinate could affect a developing fetus during pregnancy, if it passed into breast milk, or if it was safe for a breastfed infant.
People who were pregnant, planning to become pregnant, or planning to breastfeed should have talked to a doctor before taking Recombinate. The medication should only have been administered in these circumstances if the potential benefits to the mother clearly outweighed the potential risks to the developing fetus or infant.
Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
