Study identification

EU PAS number

EUPAS47916

Study ID

48479

Official title and acronym

A Prospective, Observational, Post-Authorisation Efficacy Study to Assess Long-term Effectiveness of Risdiplam in Patients with Genetically Confirmed 5q SMA

DARWIN EU® study

No

Study countries

Austria
Germany
Sweden
Switzerland

Study description

This study is a multi-country, non-interventional, longitudinal cohort study utilising data from existing SMA patient registries. The study was initially proposed to be conducted using both primary data collection and secondary data extracted from existing SMA registries. However, given the feasibility assessment results, the prospective primary
data collection was removed from the initial version of the plan, after discussion with CHMP. This study is a multi-country, non-interventional, longitudinal cohort study utilising data from existing SMA patient registries. The study was initially proposed to be conducted using both primary data collection and secondary data extracted from existing SMA registries. However, given the feasibility assessment results, the prospective primary data collection was removed from the initial version of the plan, after discussion with CHMP.

Study status

Ongoing
Research institutions and networks

Institutions

PPD Evidera
Sweden
United Kingdom
United States
First published:
22/09/2025
Institution Laboratory/Research/Testing facility Non-Pharmaceutical company ENCePP partner
Pediatric Neuromuscular Clinical Research (PNCR)

Contact details

Nahila Justo

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Hoffmann-La Roche
Regulatory

Was the study required by a regulatory body?

Yes

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

Not applicable