Study identification

PURI

https://redirect.ema.europa.eu/resource/50547

EU PAS number

EUPAS34611

Study ID

50547

Official title and acronym

Drug utilisation study of Eliglustat for the treatment of Gaucher Disease Type 1 in Europe using electronic healthcare records (ELIGLC06913)

DARWIN EU® study

No

Study countries

Belgium
France
Germany

Study description

Eliglustat (Cerdelga®) is an oral treatment indicated for the long-term treatment of Gaucher Disease Type 1 (GD1). This treatment was approved by the EMA on 19/01/2015. Today, the EMA requested a drug utilisation study (DUS) on Eliglustat and its concomitant treatments in Europe. Eliglustat is metabolised primarily by CYP2D6, and to a lesser extent by CYP3A. The concomitant use of drugs affecting CYP2D6 or CYP3A4 activity may alter Eliglustat plasma concentrations. Inversely, Eliglustat may alter the activity of these substances. Therefore, it is important that the use of inhibitors of CYP2D6 and/or CYP3A, strong CYP3A inducers, P-gp and CYP2D6 substrates as concomitant medications among patients treated with Eliglustat be carefully monitored. Moreover, a pilot study conducted in Lombardia (Italy) in 2015 showed that a lot of patients with GD were treated with several concomitant medications such as inhibitors of CYP2D6 and of CYP3A. Consequently, this DUS would inform on the usage patterns prevailing in Europe and evaluate the effectiveness of risk minimisation measures that have been put in place. The aim of the DUS is to estimate the dose and duration of Eliglustat therapy as well as the proportion, type, and duration of past and concomitant medication use in GD1 patients treated with Eliglustat. The concomitant medications of interest are strong and moderate inhibitors of CYP2D6 and/or CYP3A inhibitors, strong CYP3A inducers, P-gp substrates and CYP2D6 substrates. Also, the DUS will describe the healthcare service pattern for the prescriptions of concomitant medications in patients treated with Eliglustat. The DUS is planned in five European countries (Belgium, France, Denmark, Germany and UK) using existing health databases. In each country, the period of study will be from one year before the launching of Eliglustat to Q3 2022. The final report to EMA is planned in Q4 2024.

Study status

Planned
Research institutions and networks

Institutions

Parexel International
United States
First published:
10/12/2024
InstitutionNon-Pharmaceutical companyENCePP partner

Contact details

Katja M Hakkarainen

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:

Data analysis start date

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Sanofi-Genzyme
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)