Study identification

PURI

https://redirect.ema.europa.eu/resource/46830

EU PAS number

EUPAS13350

Study ID

46830

Official title and acronym

SCENESSE® (Afamelanotide 16mg) Retrospective Chart Review (SCENESSE® Chart Review)

DARWIN EU® study

No

Study countries

Austria
Netherlands

Study description

In December 2014, marketing authorization for SCENESSE® was granted in Europe under exceptional circumstances (Article 14(8) of Regulation (EC) No 726/2004). To address the missing long-term safety data, the establishment of a Disease Registry was imposed as a specific obligation with data to be collected from both EPP patients and physicians. In addition, there was an obligation that the MAH should also undertake a retrospective chart review study (RCR).
On 11 April 2024, a positive opinion was received from the EMA to terminate the obligation to conduct a RCR study. This was as a consequence of no patient enrolment in the study while comprehensive scientific data on long-term safety and outcome endpoints is being generated through the specific obligation of the EPP Disease Registry (EUPAS13001/study ID 46824 and EUPAS13004/study ID 46827). The discontinuation of the RCR study will not impact the benefit-risk profile of SCENESSE® as data continues to be obtained from the Disease Registry which remains positive. Therefore the RCR study has been cancelled and will not be conducted.

Study status

Planned
Research institutions and networks

Institutions

Networks

European Porphyria Initiative

Contact details

Pilar Bilbao

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:

Data analysis start date

Planned:

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

Clinuvel
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)