Pulmonary exacerbations and bacterial Routine pharmacovigilance; sputum colonisation with long-term Study 105, two-year follow-up from ivacaftor treatment. Study 102, 103, 104; An observational to evaluate longterm, safety. Use in children between 6 and 11 years Routine pharmacovigilance; of age Study 105, two-year follow-up from Study 102, 103, 104, Study 110, Study 111, Study 112; Analysis of PK data from studies on the need to perform a dose-finding study in children 6 to 11 years of age; An observational study to evaluate longterm, safety. Reconciliation of issues outlined in the RMP report Table 7 summarises the recommendation of the RMP evaluation report and the sponsor’s responses to the issues raised. Table 7: Reconciliation issues outlined in the RMP Evaluation Report Recommendation in RMP Sponsor’s response RMP evaluation report evaluator’s comment 1. Safety considerations may be Vertex has the The raised by the non-clinical and clinical comments of the sponsor’s evaluators through the consolidated nonclinical and clinical response is section 31 request and/or the evaluators into satisfactory. nonclinical and clinical evaluation consideration and there are reports respectively. It is important no resulting changes to the to ensure that the information RMP. provided in response to these includes a consideration of the relevance for the RMP, and any specific information needed to address this issue in the RMP. For any safety considerations so raised, the sponsor should provide information that is relevant and necessary to address the issue in the RMP. 2. The sponsor should provide an An updated foreign The update to the status of its overseas regulatory status was evaluator applications. Explanations need to be submitted. No applications has noted provided for any decision of have been rejected, the update of rejection, deferral or withdrawal. deferred or withdrawn overseas internationally with respect regulatory status.
Pulmonary exacerbations and bacterial sputum colonisation with long-term ivacaftor treatment. Routine pharmacovigilance; Study 105, two-year follow-up from Study 102, 103, 104; An observational to evaluate longterm, safety. Use in children between 6 and 11 years of age Routine pharmacovigilance; Study 105, two-year follow-up from Study 102, 103, 104, Study 110, Study 111, Study 112; Analysis of PK data from studies on the need to perform a dose-finding study in children 6 to 11 years of age; An observational study to evaluate longterm, safety. Reconciliation of issues outlined in the RMP report Table 7 summarises the recommendation of the RMP evaluation report and the sponsor’s responses to the issues raised. Table 7: Reconciliation issues outlined in the RMP Evaluation Report Recommendation in RMP evaluation report Sponsor’s response RMP evaluator’s comment 1. Safety considerations may be raised by the non-clinical 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 these includes a consideration of the relevance for the RMP, and any specific information needed to address this issue in the RMP. For any safety considerations so raised, the sponsor should provide information that is relevant and necessary to address the issue in the RMP. Vertex has the comments of the nonclinical and clinical evaluators into consideration and there are no resulting changes to the RMP. The sponsor’s response is satisfactory. 2. The sponsor should provide an update to the status of its overseas applications. Explanations need to be provided for any decision of rejection, deferral or withdrawal. An updated foreign regulatory status was submitted. No applications have been rejected, deferred or withdrawn internationally with respect The evaluator has noted the update of overseas regulatory status.
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