What is Replagal?

Replagal is medicine that contains the active substance agalsidase alfa. It is available as a concentrate to be made into solution for infusion (drip) into a vein. What is Replagal used for? Replagal is used to treat patients who have Fabry disease, a rare inherited disorder.

Who makes Replagal?

Replagal is an enzyme replacement therapy (ERT) for people with Fabry disease, providing them with the functional enzyme they are missing in a form that their body can use. It is being developed by the biotechnology company Shire (now owned by Takeda).

How often should I take Fabrazyme or Replagal?

Fabrazyme is a lyophylized powder in 35-mg vials that are reconstituted and diluted prior to infusion at a dose of 1 mg/kg every 2 weeks. Replagal is provided as liquid formulation at a concentration of 1 mg/ml in 3.5-ml vials that are diluted prior to infusion at 0.2 mg/kg every 2 weeks.

How are Fabrazyme and Replagal enzyme preparations produced?

Although both enzyme preparations are produced from the same cDNA sequence by genetic modification of mammalian cells, the cell types used are different. Fabrazyme is produced in a CHO cell line, and Replagal is produced in a human cell line of undisclosed origin.

Are there any side effects to the injection of Fabrazyme?

For injection: 5 mg or 35 mg of agalsidase beta as a white to off-white, lyophilized cake or powder in a single-dose vial for reconstitution. None. In clinical trials and postmarketing safety experience with Fabrazyme, approximately 1% of patients developed anaphylactic or severe hypersensitivity reactions during Fabrazyme infusion.

What is the infusion rate for Fabrazyme rechallenge?

The initial two rechallenge doses of Fabrazyme were administered as a 0.5 mg/kg dose per week at an initial infusion rate of 0.01 mg/min for the first 30 minutes (1/25 th the usually recommended maximum infusion rate).