Regulatory Decision Summary for ADYNOVATE

Clinical studies with Adynovate have shown that it is an effective Factor VIII replacement therapy for prevention and treatment of bleeding episodes and perioperative management. Pharmacokinetic studies show that PEGylation results in a product that has a longer half-life than that of Advate (1.4-1.5 fold) resulting in the potential for less frequent FVIII infusions for some patients and possible increased compliance with a prophylactic schedule. The Adynovate pivotal study supports prophylactic treatment over on-demand treatment since there is a lower annualized bleeding rate and fewer bleeding episodes with potentially less joint damage.

Non-clinical studies have not shown any toxicity or unexpected immunogenicity for either Adynovate or PEG alone. PEGylated products have been used for many years. The size of the PEG molecule used in Adynovate has not been associated with adverse events. There were no unexpected adverse events in clinical studies and no neutralizing antibodies (inhibitors) have developed thus far. It is unknown at this time if long term Adynovate will result in a decreased or increased risk of inhibitor formation. Follow up will include Periodic Safety Update Reports (PSURs) and Periodic Benefit-Risk Evaluation reports (PBRERs) which will provide continuing safety assessment. Adynovate has a favourable benefit/risk profile at this time and was recommended for approval.

For more information on Health Canadas decision, please view the Summary Basis of Decision.