Patients be informed of risks in self-administration: CDSCO Panel tells Takeda Biopharm on Agalsidase Alfa
New Delhi: In line with the proposal presented by Takeda Biopharmaceuticals for an update in the package insert regarding home infusion for Agalsidase Alfa (REPLAGAL) indicated for the treatment of enzyme replacement therapy for Fabry Disease, the Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organization (CDSCO) has opined that the drug should preferably be administered by the treating physician in clinical settings but in case of inability, the drug can also be administered with the help of trained paramedical staff at home after obtaining consent from the patient and with the approval of the treating physician only.
In addition, the expert panel suggested that patients be informed through the Informed Consent Form (ICF) of the risks involved in the administration of drugs in home settings.
This came after Takeda Biopharmaceuticals presented the proposal for an update in the package insert regarding home infusion and administration by the patient in the presence of a responsible adult or administration by the patient’s caregiver (self-administration) for patients who are tolerating their infusions well.
The Committee noted that the drug is indicated for the treatment of enzyme replacement therapy for Fabry disease, and proposed changes in package inserts are approved by EMA (European Medicines Agency).
Fabry disease is a multi-systemic, X-linked lysosomal storage disease caused by decreased activity of alpha-galactosidase A and results in lysosomal accumulations of neutral glycosphingolipids and globotriaosylceramide GL-3.
Agalsidase alfa is a recombinant human alpha-galactosidase indicated to treat Fabry disease, a genetic deficiency in the enzyme leading to the buildup of globotriaosylceramide.
α-galactosidase A is uptaken by cells via the mannose-6 phosphate receptor. Agalsidase alfa hydrolyzes globotriaosylceramide and other glycosphingolipids that would normally be hydrolyzed by endogenous α-galactosidase A. Preventing the accumulation of glycosphingolipids prevents or reduces the severity of manifestations of Fabry disease such as renal failure, cardiomyopathy, or cerebrovascular events.
At the recent SEC meeting for Endocrinology and Metabolism, the expert panel reviewed the proposal presented by Takeda Biopharmaceuticals for an update in the package insert of Agalsidase Alfa (REPLAGAL) regarding home infusion and administration by the patient in the presence of a responsible adult or administration by the patient’s caregiver (self-administration) for patients who are tolerating their infusions well.
After detailed deliberation, the committee recommended that the drug should preferably be administered by the treating physician in the clinical setting but in case of inability, the drug can also be administered with the help of trained paramedical staff at home after obtaining consent from the patient and with the approval of the treating physician only.
Furthermore, the expert panel added that patients shall be informed through the Informed Consent Form (ICF) of the risks involved in the administration of drugs in home settings.
'ICF shall be obtained by the treating physician. ICF format with standard language shall be prepared by the MA (marketing authorization) holder, who will provide the same to the treating physician," the committee added.
