when this document is attachment in the next updated. revised ASA for taliglucerase alfa, once finalised. The sponsor also commits to updating the ASA in line with the availability of the revised EU-RMP, estimated to be available in December 2013. Another post-marketing The sponsor advised This is acceptable requirement to the US that PB-06-002 is FDA was to complete the now complete and the ongoing trial PB-06-002, Clinical Study Report is entitled Multicenter, provided for assessment “A Open-label, Switchover of safety data only. to Assess the Safety Additional studies are and Efficacy of also underway in which safety data with Taliglucerase alfa in Patients with Gaucher relevance to the RMP Disease Treated with will be collected. A final Imiglucerase (Cerezyme) protocol for Study Enzyme Replacement B3031002 and synopses Therapy.” This trial will for Studies PB-06-006 obtain safety and and PB-06-007 have efficacy data in adult and been provided. paediatric patients with The post-marketing Type 1 GD, including registry protocol data on allergic and (B3031002) is now immune-mediated finalised and will be reactions, and included in the RMP unexpected risks from pharmacovigilance plan antibody development. and ASA. The sponsor should The ongoing clinical provide compelling Studies PB-06-006 and justification as to why PB-06-007 are extension this ongoing study has studies in paediatric and not been included in the adults, respectively. pharmacovigilance plan. These studies have also Alternatively the been included in the sponsor may include this pharmacovigilance plan. ongoing study in the pharmacovigilance plan and amend the ASA accordingly. The sponsor should The sponsor provided a This is acceptable. provide a tabular tabular summary of the Risk including columns ‘Summary Management Plan in addressing each Australia’ in a revised individual safety ASA, including reference concern, the proposed to specific routine risk pharmacovigilance
when this document is next updated. attachment in the revised ASA for taliglucerase alfa, once finalised. The sponsor also commits to updating the ASA in line with the availability of the revised EU-RMP, estimated to be available in December 2013. Another post-marketing requirement to the US FDA was to complete the ongoing trial PB-06-002, entitled “A Multicenter, Open-label, Switchover to Assess the Safety and Efficacy of Taliglucerase alfa in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy.” This trial will obtain safety and efficacy data in adult and paediatric patients with Type 1 GD, including data on allergic and immune-mediated reactions, and unexpected risks from antibody development. The sponsor should provide compelling justification as to why this ongoing study has not been included in the pharmacovigilance plan. Alternatively the sponsor may include this ongoing study in the pharmacovigilance plan and amend the ASA accordingly. The sponsor advised that PB-06-002 is now complete and the Clinical Study Report is provided for assessment of safety data only. Additional studies are also underway in which safety data with relevance to the RMP will be collected. A final protocol for Study B3031002 and synopses for Studies PB-06-006 and PB-06-007 have been provided. The post-marketing registry protocol (B3031002) is now finalised and will be included in the RMP pharmacovigilance plan and ASA. The ongoing clinical Studies PB-06-006 and PB-06-007 are extension studies in paediatric and adults, respectively. These studies have also been included in the pharmacovigilance plan. This is acceptable The sponsor should provide a tabular ‘Summary of the Risk Management Plan in Australia’ in a revised ASA, including reference to specific routine risk The sponsor provided a tabular summary including columns addressing each individual safety concern, the proposed pharmacovigilance This is acceptable.