A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with Voxzogo® (vosoritide) (BMN 111-603)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/50251
• EU PAS number:
  EUPAS47514
• Study ID:
  50251
• Official title and acronym:
  A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with Voxzogo® (vosoritide) (BMN 111-603)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Belgium

  Czechia

  France

  Germany

  Italy

  Portugal

  Spain
• Study description:
  This post-authorisation safety study (PASS, BMN 111-603) will evaluate the long-term safety of treatment with Voxzogo in subjects with achondroplasia (ACH) in a real-world setting. This study is designed to monitor long-term safety in an observational setting in line with standard of care, with a focus on long term skeletal effects. It aims to supplement long-term safety data which is being collected in a series of ongoing interventional, clinical trials.
• Study status:
  Ongoing

Research institution and networks

Institutions

Quintiles

Study timelines

Date when funding contract was signed

Actual: 11/02/2022

Study start date

Planned: 31/03/2023

Actual: 17/04/2023

Date of final study report

Planned: 31/12/2035

Sources of funding

Other

More details on funding

BioMarin International Ltd.

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)