V. Pharmacovigilance findings Risk management plan The sponsor submitted a Risk Management Plan EU-RMP Version 1.0 (dated 26 April 2013) with an Australian Specific Annex (ASA) Version: 1.0 (dated 2 September 2013) which was reviewed by the TGA’s Office of Product Review (OPR). Safety specification The sponsor provided a summary of ongoing safety concerns which are shown in Table 12. Table 12. Summary of ongoing safety concerns Pharmacovigilance plan The sponsor proposes routine pharmacovigilance activities to monitor all the specified ongoing safety concerns, including targeted questionnaires for the important potential risk: Cord Compression (including laxity and unmasking myelopathic ‘Spinal/Cervical symptoms)’ and the important missing information: Experience (specifically ‘Subgroup pregnant/lactating women)’. Additional pharmacovigilance activities are proposed to further monitor and characterise all the specified ongoing safety concerns. Risk minimisation activities The sponsor has concluded that routine risk minimisation activities for all the specified ongoing safety concerns are sufficient, except for the important missing information: ‘Size of safety database’ for which no routine risk minimisation activities are proposed; and for the important identified risk: reactions (including anaphylaxis and severe allergic ‘Infusion reactions)’ for which additional risk minimisation activities in the form of healthcare provider educational materials are also proposed. Issues surrounding the proposed risk minimisation activities are discussed in Table 13 below. Reconciliation of issues outlined in the RMP report Table 13 summarises the OPR’s first round evaluation of the RMP, the sponsor’s responses to issues raised by the OPR and the OPR’s evaluation of the sponsor’s responses. Table 13. Reconciliation of issues outlined in the RMP report Recommendation in RMP Sponsor’s response OPR evaluation report evaluator’s comment Safety considerations may be raised The sponsor states: on This is ‘Based by the nonclinical and clinical the review, no new safety acceptable. evaluators through the consolidated consideration has been raised. section 31 request and/or the BioMarin will continue to Nonclinical and Clinical Evaluation review safety information Reports respectively. It is important related to Vimizim, and for any to ensure that the information new safety considerations provided in response to include a raised, BioMarin will address consideration of the relevance for the
V. Pharmacovigilance findings Risk management plan The sponsor submitted a Risk Management Plan EU-RMP Version 1.0 (dated 26 April 2013) with an Australian Specific Annex (ASA) Version: 1.0 (dated 2 September 2013) which was reviewed by the TGA’s Office of Product Review (OPR). Safety specification The sponsor provided a summary of ongoing safety concerns which are shown in Table 12. Table 12. Summary of ongoing safety concerns Pharmacovigilance plan The sponsor proposes routine pharmacovigilance activities to monitor all the specified ongoing safety concerns, including targeted questionnaires for the important potential risk: ‘Spinal/Cervical Cord Compression (including laxity and unmasking myelopathic symptoms)’ and the important missing information: ‘Subgroup Experience (specifically pregnant/lactating women)’. Additional pharmacovigilance activities are proposed to further monitor and characterise all the specified ongoing safety concerns. Risk minimisation activities The sponsor has concluded that routine risk minimisation activities for all the specified ongoing safety concerns are sufficient, except for the important missing information: ‘Size of safety database’ for which no routine risk minimisation activities are proposed; and for the important identified risk: ‘Infusion reactions (including anaphylaxis and severe allergic reactions)’ for which additional risk minimisation activities in the form of healthcare provider educational materials are also proposed. Issues surrounding the proposed risk minimisation activities are discussed in Table 13 below. Reconciliation of issues outlined in the RMP report Table 13 summarises the OPR’s first round evaluation of the RMP, the sponsor’s responses to issues raised by the OPR and the OPR’s evaluation of the sponsor’s responses. Table 13. Reconciliation of issues outlined in the RMP report Recommendation in RMP evaluation report Sponsor’s response OPR evaluator’s comment Safety considerations may be raised by the nonclinical and clinical evaluators through the consolidated section 31 request and/or the Nonclinical and Clinical Evaluation Reports respectively. It is important to ensure that the information provided in response to include a consideration of the relevance for the The sponsor states: ‘Based on the review, no new safety consideration has been raised. BioMarin will continue to review safety information related to Vimizim, and for any new safety considerations raised, BioMarin will address This is acceptable.