The sponsor has not provided study protocols or protocol synopses for all studies referenced in the pharmacovigilance plan. The sponsor should provide the missing study protocols or protocol synopses as soon as they become available. With regard to references made to additional pharmacovigilance activities in the submitted EU-RMP, the sponsor has not provided consistent study name labelling or protocol number labelling throughout the document; for example the nonclinical placental and lactation study/studies is/are referred to by different names and it remains unclear whether the same study is meant. The sponsor should update the RMP document and refer to the same studies by the same name throughout. Risk minimisation activities The sponsor proposes routine and additional risk minimisation activities. Reconciliation of issues outlined in the RMP report Table 12 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 12: Reconciliation of issues outlined in the First Round RMP Evaluation Report Recommendation in RMP Sponsor’s response/summary of the OPR evaluator’s evaluation report response comment 1. Safety considerations Sponsor confirms that the The sponsor’s response ‘The may be raised by the Nonclinical and Clinical Evaluation has been noted. nonclinical and clinical Reports have been reviewed to ensure evaluators through the that any responses provided to issues consolidated section 31 raised have been considered for request and/or the relevance to the Risk Management Nonclinical and Clinical Plan.’ Evaluation Reports respectively. It is important to ensure that the information provided in response to includes a consideration of the relevance for the Risk Management Plan, and any specific information needed to address this issue in the RMP. For any safety considerations so raised, please provide information that is relevant and necessary to address the issue in the RMP. 2. The sponsor should is formulated as a hard The sponsor’s response ‘Cerdelga clarify the above and in capsule for oral administration has been noted. particular should provide a containing 84 mg eliglustat which is The sponsor has not compelling justification for equivalent to 100 mg eliglustat specified which the following: tartrate. Information on the dose documents support an conversion for the salt was included as immediate dose of 100 part of the cover letter for the
The sponsor has not provided study protocols or protocol synopses for all studies referenced in the pharmacovigilance plan. The sponsor should provide the missing study protocols or protocol synopses as soon as they become available. With regard to references made to additional pharmacovigilance activities in the submitted EU-RMP, the sponsor has not provided consistent study name labelling or protocol number labelling throughout the document; for example the nonclinical placental and lactation study/studies is/are referred to by different names and it remains unclear whether the same study is meant. The sponsor should update the RMP document and refer to the same studies by the same name throughout. Risk minimisation activities The sponsor proposes routine and additional risk minimisation activities. Reconciliation of issues outlined in the RMP report Table 12 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 12: Reconciliation of issues outlined in the First Round RMP Evaluation Report Recommendation in RMP evaluation report Sponsor’s response/summary of the response OPR evaluator’s comment 1. 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 includes a consideration of the relevance for the Risk Management Plan, and any specific information needed to address this issue in the RMP. For any safety considerations so raised, please provide information that is relevant and necessary to address the issue in the RMP. ‘The Sponsor confirms that the Nonclinical and Clinical Evaluation Reports have been reviewed to ensure that any responses provided to issues raised have been considered for relevance to the Risk Management Plan.’ The sponsor’s response has been noted. 2. The sponsor should clarify the above and in particular should provide a compelling justification for the following: ‘Cerdelga is formulated as a hard capsule for oral administration containing 84 mg eliglustat which is equivalent to 100 mg eliglustat tartrate. Information on the dose conversion for the salt was included as part of the cover letter for the The sponsor’s response has been noted. The sponsor has not specified which documents support an immediate dose of 100
auspar-eliglustat-150818.docx