PEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers.
Haemophilia
; 19(1): 11-20, 2013 Jan.
Article
in En
| MEDLINE
| ID: mdl-22913770
ABSTRACT
PEGylation is the technology involving the covalent attachment of polyethylene glycol (PEG) to a protein-, peptide- or small-molecule drug to improve their pharmacokinetic, pharmacodynamic and immunological profiles, and thus, enhance the therapeutic effect. Today, PEGylation of proteins is a well-established technology and is being used in the treatment of a variety of clinical disorders. Several PEGylated coagulation proteins for haemophilia A and B are under development with the goal of prolonging the circulation half-life of factor VIII (FVIII) or factor IX. The prolongation of half-life, resulting in less frequent injections can provide significant benefits in improving the quality of life of subjects with haemophilia and improvement in adherence to treatment. A review of published literature on PEGylated therapeutic products currently approved for human use and a discussion of a PEGylated recombinant FVIII molecule (BAY 94-9027, Bayer HealthCare, Berkeley, CA, USA) currently being investigated in the pivotal clinical trial prior to registration is provided. Available safety information of PEGylated proteins containing high molecular weight PEG does not indicate any safety concerns to date, following long-term (chronic) use in animal models or patients. Chronic use of currently available PEGylated products has been shown to be safe, paving the way for chronic use of PEGylated coagulation products in persons with haemophilia.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Polyethylene Glycols
/
Factor IX
/
Factor VIII
/
Hemophilia B
/
Hemophilia A
Type of study:
Prognostic_studies
Aspects:
Patient_preference
Limits:
Humans
Language:
En
Journal:
Haemophilia
Journal subject:
HEMATOLOGIA
Year:
2013
Document type:
Article
Affiliation country:
United States