Use (ATU) reports identified as of post-marketing experience”, but actually reports “reports of patients treated in France under a Compassionate Use Program (CUP); literature references. Module 1: Application letter; application forms; proposed Australian Product Information (PI); proposed Australian Consumer Medicine Information (CMI); information about the sponsor’s experts; orphan drug designation; details of compliance with meetings and submission processes; overseas regulatory status including draft US and European prescribing information documents; summary of the BA/BE study; Risk Management Plan (RMP) for Australia. Module 2: Quality overall summary; Nonclinical overview; Clinical overview; Summary of biopharmaceutics studies and associated analytical methods; Summary of clinical pharmacology studies; Summary of clinical efficacy; Summary of clinical safety; Literature references; Synopses of individual studies. Paediatric data The pivotal Phase II/III clinical and efficacy study (201) was undertaken in infants with proliferating IH aged from 30 to 150 days. The proposed indication for Hemangiol is applicable only to infants in this age group with proliferating IH requiring systemic therapy. Good clinical practice The studies forming the sponsor’s clinical development program for the proposed product for the proposed indication were undertaken in compliance with the principles of good clinical practice. Pharmacokinetics Studies providing pharmacokinetic data The included two studies providing pharmacokinetic (PK) data (study V004 SB 101 2A; study V00400 SB 1 02); these two studies will be referred to as study 1012A and study 102, respectively, in this Clinical Evaluation Report (CER). In addition to a non-compartmental PK analysis in infants with IH, study 102 also included a population pharmacokinetic (PPK) analysis in this patient group identified as V0400 101 and referred to as the PPK report in this CER. The two studies (including the PPK report) have been evaluated and relevant data from these studies (including the PPK report) have been included in the CER (text and supporting tables and figures). Studies 1012A, 102 and the PPK report are briefly outlined below (Table 1). Table 1. Brief outline of the studies providing pharmacokinetic data. Study PK topic Population (n) Primary objective Treatments
Use (ATU) reports identified as “reports of post-marketing experience”, but actually reports of patients treated in France under a Compassionate Use Program (CUP); literature references. Module 1: Application letter; application forms; proposed Australian Product Information (PI); proposed Australian Consumer Medicine Information (CMI); information about the sponsor’s experts; orphan drug designation; details of compliance with meetings and processes; overseas regulatory status including draft US and European prescribing information documents; summary of the BA/BE study; Risk Management Plan (RMP) for Australia. Module 2: Quality overall summary; Nonclinical overview; Clinical overview; Summary of biopharmaceutics studies and associated analytical methods; Summary of clinical pharmacology studies; Summary of clinical efficacy; Summary of clinical safety; Literature references; Synopses of individual studies. Paediatric data The pivotal Phase II/III clinical and efficacy study (201) was undertaken in infants with proliferating IH aged from 30 to 150 days. The proposed indication for Hemangiol is applicable only to infants in this age group with proliferating IH requiring systemic therapy. Good clinical practice The studies forming the sponsor’s clinical development program for the proposed product for the proposed indication were undertaken in compliance with the principles of good clinical practice. Pharmacokinetics Studies providing pharmacokinetic data The submission included two studies providing pharmacokinetic (PK) data (study V004 SB 101 2A; study V00400 SB 1 02); these two studies will be referred to as study 1012A and study 102, respectively, in this Clinical Evaluation Report (CER). In addition to a non-compartmental PK analysis in infants with IH, study 102 also included a population pharmacokinetic (PPK) analysis in this patient group identified as V0400 101 and referred to as the PPK report in this CER. The two studies (including the PPK report) have been evaluated and relevant data from these studies (including the PPK report) have been included in the CER (text and supporting tables and figures). Studies 1012A, 102 and the PPK report are briefly outlined below (Table 1). Table 1. Brief outline of the studies providing pharmacokinetic data. Study PK topic Population (n) Primary objective Treatments