Factor IX (Human), Factor IX Complex (Human) and Laronidase

• Generic Name: coagulation factor ix
• Dosage Forms: n.a.
• Other Brand Names: Alphanine SD, Alprolix, BeneFIX, Idelvion, Ixinity, Mononine, Rebinyn, Rixubis, BeneFIX 250 Int'l Units, BeneFIX

Prevention and control of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease).

Maintenance of hemostasis in patients with hemophilia B undergoing surgery.

In patients with preexisting thromboembolic risk factors, some experts state that pure (i.e., single-factor) factor IX preparations are preferred over factor IX complex for treatment of hemophilia B. (See Thromboembolic Events under Cautions.)

Also used for routine prophylaxis (i.e., administration at regular intervals on an ongoing basis) to prevent or reduce frequency of hemorrhagic events. Such prophylactic therapy currently considered the standard of care for patients with hemophilia B. Decreases frequency of spontaneous musculoskeletal hemorrhage, preserves joint function, and improves quality of life.

Several factor IX concentrates are currently available in the US, including a variety of recombinant and plasma-derived preparations; the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation recommends preferential use of recombinant factor IX preparations because of their potentially superior safety profile with respect to pathogen transmission. (See Risk of Transmissible Agents in Plasma-derived Preparations under Cautions.) Other experts (e.g., World Federation of Hemophilia) state that choice of preparation should be determined by local criteria. When selecting an appropriate factor IX preparation, consider characteristics of each clotting factor concentrate, individual patient variables, patient/provider preference, and emerging data.

Factor IX (human) is not indicated for treatment of coagulation factor II, VII, or X deficiency or for management of hemophilia A in patients with inhibitors to factor VIII.

Factor IX complex (human) is not indicated for treatment of factor VII deficiency or other coagulation factor deficiencies.

Factor IX complex (human) (i.e., 3-factor PCC) has been used for urgent reversal of warfarin anticoagulation in patients experiencing major bleeding or in those who require immediate reversal of anticoagulation for other reasons (e.g., urgent surgery).

Factor IX (human) is not indicated for treatment or reversal of coumarin-induced anticoagulation or for treatment of hemorrhagic states caused by hepatitis-induced lack of production of liver-dependent coagulation factors.

• Generic Name: laronidase
• Dosage Forms: n.a.
• Other Brand Names: Aldurazyme

Laronidase contains an enzyme that occurs naturally in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Laronidase helps replace this missing enzyme in such people.

Laronidase is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis) or MPS I, also called Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome.

MPS I is a metabolic disorder in which the body lacks the enzyme needed to break down certain substances. These substances can build up in the body, causing enlarged organs, abnormal bone structure, changes in facial features, breathing problems, heart problems, vision or hearing loss, and changes in mental or physical abilities.

Laronidase may improve breathing and walking ability in people with this condition. However, this medication is not a cure for MPS I.

Laronidase may also be used for purposes not listed in this medication guide.