Helixate^® FS

Antihemophilic Factor (Recombinant) Formulated with Sucrose

Q. Are there any contraindications for Helixate^® FS?

A. Known intolerance or allergic reactions to constituents of the preparation or known allergies to hamster and mouse proteins may be a contraindication for Helixate^® FS. Baby hamster kidney (BHK) cells are used in the fermentation process to grow the recombinant factor VIII. Mouse antibody proteins (monoclonal antibody immunoaffinity chromatography) are used in the purification process to remove factor VIII from the fermentation medium.

Q. Does Helixate^® FS contain albumin?

A. Unlike many other factor VIII concentrates (both plasma-derived and recombinant), Helixate^® FS uses sucrose instead of albumin in the final product to stabilize the recombinant factor VIII molecule. However, human albumin is needed to protect and stabilize the recombinant factor VIII protein that is being secreted by the baby hamster kidney cells in the fermentation medium. Most of the albumin is removed during the many purification steps, but a minimal amount of albumin (.007 milligrams/mL) does remain in the product. That is 1000 times less albumin than original Helixate^® Antihemophilic Factor (Recombinant). Albumin is not added during the purification or formulation steps.

Q. Does the cell culture medium contain any bovine-derived products?

A. The cell culture medium does not contain any proteins derived from animal sources. For example, bovine insulin has been replaced with recombinant insulin. However, human plasma protein solution is still added to the cell culture medium.

Q. How should Helixate^® FS be stored?

A. When stored at refrigerator temperature, 2°-8°C (36°-46°F), Helixate^® FS is stable for the period indicated by the expiration date on the label. Storage of lyopholized powder at room temperature (up to 25°C or 77°F) for 3 months, such as in home treatment situations, may be done. Avoid freezing. Protect from extreme exposure to light.

Q. Is Helixate^® FS a recombinant product?

A. Helixate^® FS is a recombinant factor VIII product that is manufactured using a baby hamster kidney (BHK) cell line. In the purification and formulation steps, the human albumin used as a factor stabilizer in original Helixate^® Antihemophilic Factor (Recombinant) has been replaced with sucrose in Helixate^® FS; however, a highly purified plasma protein is still used in the fermentation medium.

Q. Is Helixate^® FS effective?

A. Yes. Helixate^® FS has similar biologic activity to original Helixate^® Antihemophilic Factor (Recombinant) and to factor VIII from human plasma. In clinical studies involving surgeries for previously treated and untreated patients, 100% of responses were rated excellent/good with Helixate^® FS. In clinical studies involving previously treated and untreated patients, 93.5% and 90%, respectively, of the bleeding episodes resolved with one or two treatments with Helixate^® FS and response to treatment was rated as excellent/good in 80.5% and 90%, respectively.

Q. Is Helixate^® FS safe?

A. Yes. In clinical studies involving previously treated patients, no inhibitor formation was detected. In clinical studies involving previously untreated patients, 15% developed inhibitors to Helixate^® FS. There were no viral transmissions in any of the patients. Adverse events such as local injection site reaction, nausea, dizziness, and rash were reported in <1% of the patients.

Q. Is Helixate^® FS virally inactivated?

A. Helixate^® FS uses a solvent/detergent process designed to help eliminate lipid-enveloped viruses (such as HIV, hepatitis B and C, and herpes). Helixate^® FS also uses the patented monoclonal antibody (MAb) immunoaffinity chromatography purification process designed to help eliminate viruses. In this process, antibodies specific to a single target protein (such as factor VIII) are placed in a chromatography column. When the solution is passed through the column, the antibodies bind the target protein. There have been no reports of viral transmissions with Helixate^® FS to date.

Q. How soon after reconstitution must Helixate^® FS be administered?

A. Helixate^® FS should be administered within 3 hours after reconstitution because there is no preservative in the product.

Q. Other than sucrose, what is the purpose of the other excipients in the final formulation of Helixate^® FS?

A. Helixate^® FS is lyophilized, which means it is rapidly freeze-dried under high vacuum. When a solution is freeze-dried, crystals form. The sodium, chloride, and calcium chloride protect the product during the freeze-drying process, while the glycine and histidine stabilize the crystal matrix.

Important Safety Information

Helixate^® FS is a recombinant factor VIII (FVIII) product used to treat hemophilia A when there is a demonstrated deficiency of FVIII, a plasma clotting factor.

Helixate^® FS should be administered cautiously in patients with previous hypersensitivity to constituents of FVIII or known sensitivity to mouse or hamster protein.

Helixate^® FS should not be used by people with von Willebrand disease because it does not contain von Willebrand factor—a different protein associated with a different bleeding disorder. For more information about von Willebrand disease, please visit www.allaboutbleeding.com.

Adverse events are generally minor. The most common events include injection-site reactions, dizziness, and rash.