Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Scope of the study

Drug utilisation
Study drug and medical condition

Name of medicine

ESMYA

Study drug International non-proprietary name (INN) or common name

ULIPRISTAL ACETATE

Anatomical Therapeutic Chemical (ATC) code

(G03XB02) ulipristal
ulipristal

Medical condition to be studied

Uterine leiomyoma
Population studied

Age groups

  • Adult and elderly population (≥18 years)
    • Adults (18 to < 65 years)
      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)

Estimated number of subjects

1500
Study design details

Main study objective

The specific objectives of the study are to:
1. Assess the long term safety, including endometrial safety, of Esmya in standard medical practice
2. Assess prescription patterns of Esmya in standard medical practice.

Exploratory objective: Assess patients’ quality of life in a long-term treatment setting.

Data analysis plan

The majority of statistical analyses will be descriptive, reporting patient counts, means, standard deviations, medians, minima, and maxima for continuous variables (e.g. age and duration of symptomatic uterine fibroids) and frequencies and percentages for categorical variables (e.g. disease symptoms, prescription pattern, diagnostic test results). Two-sided 95% confidence intervals will be estimated as appropriate and reported along with p-values to facilitate the interpretation of the significance of the findings. These findings will be compared to published epidemiological data on pre-menopausal women with abnormal uterine bleeding (AUB), where available.Baseline data (i.e. data collected prior to the first administration of Esmya), including relevant demographic characteristics will be summarised for all treated subjects.Full details of all analysis to be carried out will be presented in the Statistical Analysis Plan (SAP).

Summary results

This was a prospective, multicentre, non-interventional study in female patients who had been diagnosed with moderate to severe symptoms of uterine fibroids and were initiating long-term treatment with Esmya (PGL14-001, PREMIUM) or patients who were previously exposed to ulipristal acetate 5 or 10 mg in the long-term Phase III studies.
Eligible, consenting patients were enrolled from approximately 100 to 150 clinical practice sites in the EU and United Kingdom and were followed for 60 months from treatment start according to the standard medical practice of their physician.
This study provided real-world data related to the longterm safety, including endometrial safety, and current prescription patterns of Esmya as treatment for symptomatic uterine fibroids from 1359 patients who had received at least one dose of Esmya/ulipristal acetate. This study also aimed to address the following safety concerns included in the current ulipristal acetate Risk Management Plan and identified:
• 7 (0.5%) patients with inappropriate management of endometrium thickening.
• 2 (0.15%) patients with suspected inappropriate diagnosis of endometrial hyperplasia.
• 11 (0.8%) patients with suspected acute uterine bleeding requiring immediate intervention. No trend could be identified for these cases.
• 104 (7.7%) patients with continuous treatment courses beyond 3 months. No safety concern was identified among these patients compared to the rest of the study population.
• No long-term effects of prolonged treatment on the endometrium (continuous course beyond 3 months or more than 4 intermittent treatment courses).
• One case of atypical hyperplasia and one case of endometrial adenocarcinoma, however, there were no cases identified with delayed diagnosis of atypical endometrial hyperplasia or adenocarcinoma.
• No identified reports of Esmya treatment having either a beneficial or adverse impact on (uterine fibroid) surgery.
No new safety concerns have been identified in this study