Regulatory information – New guidance on type II variations and marketing authorisation extensions for orphan-designated medicines

News 18/11/2016

Guidance reflects European Commission's notice on the application of the Orphan Regulation

The European Medicines Agency (EMA) has updated its post-authorisation guidance on extensions of marketing authorisations and type II variations to include further details on the information companies need to provide when submitting a type II variation or a marketing authorisation extension for an orphan-designated medicine.

This follows the publication of a new notice by the European Commission on the application of Articles 3, 5 and 7 of Regulation (EC) No 141/2000 on orphan medicinal products. In this notice, the European Commission provides guidance on the application of the Orphan Regulation, more specifically on orphan designation provisions.

The Commission guidance clarifies amongst other things that for a new therapeutic indication that falls under a previously confirmed orphan designation, EMA's Committee for Orphan Medicinal products (COMP) will have to consider whether the specific scope of the variation raises justified and serious doubts in respect to the fulfilment of the orphan-designation criteria.

This aims to ensure that the orphan marketing authorisation only covers indications that fulfil the orphan designation criteria foreseen in the Orphan Regulation.

EMA's updated post-authorisation guidance is to be followed for applications submitted as of the date of publication of the Commission's notice, i.e. 18 November 2016. Marketing authorisation holders are advised to contact the Agency in advance of their upcoming orphan extension of indication for further advice.

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