(median) PBAC score > 100 at screening cm diameter inclusive Open-label for UPA 90 days 10 mg daily 18-48 85.1% mean age 40.0 years; mean BMI 25.4; 96.0% of child-bearing potential; mean (median) PBAC score at baseline = 319 (216). myomatous uterus < 16 weeks, at least one myoma ≥ 3 cm diameter, nil larger than 10 cm, eligible for or of 3 x 90 day placebo controlled 13 weeks 5 mg (n= 96) or 10 mg (n= 98) daily 18-50 88.0% white; mean age 41.6 years; mean BMI 25.3; 92.1% of child-bearing potential; mean (median) PBAC score at baseline = 452 (361). PBAC score > 100, myomatous uterus ≤ 16 weeks gestation equivalent, at least one myoma ≥ 3 cm diameter, nil larger than 10 cm, fibroid related anaemia defined as Hb ≤ 10.2g/dL, eligible for surgery. Double double dummy, active controlled 13 5mg (n= 102) or 10 mg (n= 103) daily 18-50 Mean age 40.4 mean BMI 25.5; 96.3% of child-bearing potential; mean (median) PBAC score at baseline = 370 (282) PBAC score > 100, myomatous uterus ≤ 16 weeks gestation equivalent, at least one myoma ≥ 3 cm diameter, nil larger than 10 cm, eligible for surgery. The pictorial bleeding assessment chart (PBAC) is a validated scoring method to objectively measure menstrual blood loss, and was used as a measure of uterine bleeding course n
submission of final data Prospective (PASS) PGL10-014: A prospective multicenter noninterventional study of women treated with Esmya (ulipristal acetate) as treatment of moderate to severe symptoms of uterine fibroids. (PREMYA, category 3) Inappropriate management of endometrium thickening (unnecessary interventions or treatments) Inappropriate diagnosis of endometrial hyperplasia (mistaking PAEC for hyperplasia) Acute uterine bleeding requiring immediate intervention Drug Induced Injury (DILI) Treatment beyond three months Delayed diagnosis of atypical endometrial hyperplasia or adenocarcinoma Impact on surgery Esmya use in “real world” practice Final study report planned Q1 2016 (4 years post approval) Retrospective non interventional study (PASS) PGL11-020: Esmya prescription patterns in Europe: A retrospective drug utilisation chart review study. (PRECISE, category 3) Inappropriate management of endometrium thickening (unnecessary interventions or treatments) Inappropriate diagnosis of endometrial hyperplasia (mistaking PAEC for hyperplasia) Treatment beyond three months Impact on surgery Esmya use in “real world” practice study report planned Q4 2015
Prospective, noninterventional study (PASS) PGL14-001: A prospective, noninterventional study to evaluate the long term safety of Esmya, in particular the endometrial safety, and the current prescription and management patterns of Esmya in a long term treatment setting. (PREMIUM, category 3) Inappropriate management of endometrium thickening (unnecessary interventions or treatments) Inappropriate diagnosis of endometrial hyperplasia (mistaking PAEC for hyperplasia) Acute uterine bleeding requiring immediate intervention Drug Induced Liver Injury (DILI) Treatment beyond three months Delayed diagnosis of atypical endometrial hyperplasia or adenocarcinoma Impact on surgery Esmya use in “real world” practice Final report planned in Q1 2023 (8 years after variation approval) Clinical PGL11-024: A Phase III, extension study of repeated intermittent courses of open-label of 10 mg ulipristal acetate. (PEARL extension 2, category 3) Long-term effects of prolonged treatment on the endometrium Safety and efficacy in total eight 3-month treatment courses Final report in Q3 2015 RMP reviewer comment The existing, ongoing and planned pharmacovigilance activities are considered acceptable for the purposes of this submission. The sponsor should make the result of PEARL study (PGL11-024) available to TGA. Risk minimisation activities Sponsor’s conclusion in regard to the need for risk minimisation activities The sponsor is proposing additional risk minimisation activities.