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EMA Guidance: Procedural Advice for Orphan Medicinal Product Designation

Yesterday (20 October 2023) the European Medicines Agency released an updated guidance on "Procedural Advice for Orphan Medicinal Product Designation".

Orphan Medicine is a medicine for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare (affecting not more than five in 10,000 people in the European Union) or where the medicine is unlikely to generate sufficient profit to justify research and development costs.

Orphan medicinal product designation is the first step in obtaining these benefits. It provides several advantages to the drug's developer, including market exclusivity, protocol assistance, and reduced fees.

In this guidance, the EMA has provided detailed procedural advice to guide applicants through the designation process.

IRIS | Regulatory & Scientific Information Management Platform is a new secure online portal for sponsors to submit applications for orphan medicinal product designation and to manage post-designation activities.

The sponsor may submit an application for orphan medicinal product designation to the Agency at any stage of development of the medicinal product as long as the criteria for designation may be justified. However, the designation application must be submitted before the submission of the application for marketing authorisation.

Sponsors should follow one of the two options below:

  • Request a pre-submission meeting: If a sponsor would like to have a preliminary discussion before submitting an orphan drug application to EMA, they can request a pre-submission meeting at least two months prior to their planned submission date.

  • Submit an application without notice: It is recommended that sponsors submit their applications few days before any of the published Submission deadlines for orphan designations | European Medicines Agency ( to allow more time for the validation process and the possibility to intervene in case of technical problems

The maximum timeframe for the evaluation of a Marketing Authorisation Application under the centralised procedure is 210 days.

To know more about the General principles, Submission, Validation, Frequently asked questions & other requirements click this LINK.


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