ADAGEN® (pegademase bovine) Injection is used to treat patients afflicted with a type of Severe Combined Immunodeficiency Disease (also known as SCID or the "Bubble Boy Disease"), which is caused by the chronic deficiency of the adenosine deaminase (ADA) enzyme.  Children with SCID are born without fully functioning immune systems, which leaves them susceptible to a wide range of infectious diseases.  ADAGEN is indicated for enzyme replacement therapy for ADA deficiency in patients with severe combined immunodeficiency disease who are not suitable candidates for or who have failed bone marrow transplantation.

ADAGEN is a PEGylated version of the ADA enzyme and represents the first successful application of enzyme replacement therapy for an inherited disease.    It is a valuable life-saving treatment option that provides predictable restoration of ADA activity to ADA SCID patients.

ADAGEN can be administered in a doctor’s office or at home while under the care and direction of a physician. 

ADAGEN provides specific and direct replacement of the deficient enzyme, but it will not benefit patients with immunodeficiency due to other causes.

Designated as an Orphan Drug, ADAGEN was approved for use by the FDA in 1990 and has over 17 years of proven clinical experience.

ADAGEN® (pegademase bovine) Injection is indicated for enzyme replacement therapy for adenosine deaminase (ADA) deficiency in patients with severe combined immunodeficiency disease (SCID) who are not suitable candidates for – or who have failed – bone marrow transplantation. 

There is no evidence to support the safety and efficacy of ADAGEN as preparatory or support therapy for bone marrow transplantation. Since ADAGEN is administered by intramuscular injection, it should be used with caution in patients with thrombocytopenia and should not be used if thrombocytopenia is severe.

The optimal dosage and schedule of administration should be established for each patient. Plasma ADA activity and red cell dATP should be determined prior to treatment.  The treatment of SCID associated with ADA deficiency with ADAGEN should be monitored by measuring plasma ADA activity and red blood cell dATP levels.

The following adverse reactions were reported during clinical trials: headache in one patient and pain at the injection site in two patients.

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