PEGVALIASE

The PBAC deferred making a recommendation for listing pegvaliase for the treatment of patients with phenylketonuria (PKU) who have inadequate blood phenylalanine (Phe) control despite prior management with available treatment options including a Phe-restricted diet and sapropterin. 

The PBAC welcomed input from individuals, clinicians and groups. The PBAC acknowledged the challenges of living with PKU including the severe and lifelong burden of dietary restrictions. The PBAC also noted the cognitive impacts of high Phe levels including difficulty concentrating, reduced mental clarity, anxiety and fatigue. 
 
The PBAC acknowledged that the existing treatment, sapropterin, does not work adequately in all patients in terms of reducing blood Phe levels and improving dietary restrictions. As such, the PBAC recognised the high unmet clinical need for effective treatments in the proposed patient population.
 
The PBAC accepted that pegvaliase is more effective than a Phe-restricted diet alone at reducing blood Phe levels. However, the PBAC considered the evidence presented in the submission did not allow confidence about the extent to which pegvaliase would provide benefits compared to a Phe-restricted diet alone. This was because of limitations in the quality of clinical evidence and methodological concerns with available studies. The PBAC acknowledged reliable clinical data were difficult to obtain given the rarity of disease. The PBAC noted that pegvaliase was associated with higher rates of hypersensitivity and skin reactions compared with a Phe-restricted diet alone.
 
However, the PBAC considered it was unclear whether the sponsor’s proposed pricing structure and risk sharing arrangement (RSA) proposal would achieve a cost-effective price and adequately manage the financial risk to the Commonwealth. The deferral was to allow further consultation with the sponsor and the Department regarding how best to implement the intent of the sponsor’s requested pricing and RSA proposal.