protocol be separately separately reported in reported in any future PSUR. future PSUR’s for Carbaglu submitted in Australia. 6. The sponsor should provide The sponsor wishes to This is considered an attachment to the ASA confirm that no forthcoming acceptable. setting out all the forthcoming studies are planned for studies and the anticipated Australia. However for their submission in Appendix 2 of the ASA Australia. summarises the status of the global studies currently underway and the anticipated timeframe for the submission of interim and final data. 7. The information in the As noted above, the updated The response has been safety database is based on a RMP document has a data noted. Of note, the cut-off date of October 2011. It lock cut-off date of 31 May documents provided are is recommended that the 2014. inconsistent, and this sponsor submits more up to should be corrected prior date data. to approval. 8. The sponsor should the All cases of potential lack of In the ASA in section RMP to clarify that lack of efficacy were reviewed in ‘pharmacovigilance efficacy is addressed through the paragraph 2.6.5. Lack of practices in Australia’ the routine pharmacovigilance efficacy was considered as a sponsor describes that only. Nevertheless, this activity potential risk. This potential routine is not described in the ASA, risk was added in the RMP pharmacovigilance and the sponsor should amend paragraph 5.1 Risk activities will be carried the ASA to include details minimization measures. out to monitor the about this activity to address potential risk of of ‘lack this potential risk in Australia. efficacy’. Furthermore, the sponsor describes various activities in this section, including: A.) Raise the awareness to physicians to not cease Carbaglu use too early in the treatment of hyperammonaemia B.) Comprehensive dosage and administration instructions within the Australian PI C.) Inclusion of the Carglumic acid Responsive Test within the Dosage and Administration section of the Australian PI to allow
protocol be separately reported in any future PSUR. separately reported in future PSUR’s for Carbaglu submitted in Australia. 6. The sponsor should provide an attachment to the ASA setting out all the forthcoming studies and the anticipated for their submission in Australia. The sponsor wishes to confirm that no forthcoming studies are planned for Australia. However Appendix 2 of the ASA summarises the status of the global studies currently underway and the anticipated timeframe for the submission of interim and final data. This is considered acceptable. 7. The information in the safety database is based on a cut-off date of October 2011. It is recommended that the sponsor submits more up to date data. As noted above, the updated RMP document has a data lock cut-off date of 31 May 2014. The response has been noted. Of note, the documents provided are inconsistent, and this should be corrected prior to approval. 8. The sponsor should the RMP to clarify that lack of efficacy is addressed through routine pharmacovigilance only. Nevertheless, this activity is not described in the ASA, and the sponsor should amend the ASA to include details about this activity to address this potential risk in Australia. All cases of potential lack of efficacy were reviewed in the paragraph 2.6.5. Lack of efficacy was considered as a potential risk. This potential risk was added in the RMP paragraph 5.1 Risk minimization measures. In the ASA in section ‘pharmacovigilance practices in Australia’ the sponsor describes that routine pharmacovigilance activities will be carried out to monitor the potential risk of ‘lack of efficacy’. Furthermore, the sponsor describes various activities in this section, including: A.) Raise the awareness to physicians to not cease Carbaglu use too early in the treatment of hyperammonaemia B.) Comprehensive dosage and administration instructions within the Australian PI C.) Inclusion of the Carglumic acid Responsive Test within the Dosage and Administration section of the Australian PI to allow