The European Medicines Agency (EMA) has updated its post-authorization guidance on how extensions of marketing authorizations and type II variations may impact orphan-designated medicines.
The updates follow 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.
EMA says that under the EC guidance, 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 fulfillment of the orphan-designation criteria. EMA’s updated guidance is to be followed for applications submitted as of the date of publication of the commission’s notice, which was 18 November 2016.
Type II Variations & Marketing Extensions
In both the Q&A guidance documents on the type II variations and the extension of marketing authorizations, EMA has revised the sections on “How shall I present my type-II-variation application?” and added two new sections on “Do I need to confirm the maintenance of my orphan designation when applying for a type-II variation?” and “Can a new indication based on less comprehensive data be added to an already authorized medicinal product?”
EMA clarifies that for a “new therapeutic indication falling within a new orphan designation [bold is EMA’s], i.e. an orphan designation other than the one(s) related to the already approved indication(s), the COMP will have to confirm the maintenance of the orphan designation before authorization of the new indication. In this case, the sponsor should provide at the time of submission a maintenance report using the template provided on the EMA website.”
Similarly, for a new therapeutic indication falling “within an already authorized orphan designation [bold is EMA’s], the COMP will have to consider if the specific scope of the variation raises justified and serious doubts in respect to the fulfillment of the orphan designation criteria and indicate if a formal review process of the maintenance of the orphan designation is needed.”
On the question of less comprehensive data, EMA notes the various pathways where less comprehensive data than normally required can be used, (i.e. a conditional marketing authorization or marketing authorization under exceptional circumstances) but granting these types of authorizations “is only foreseen in the context of an application for an initial marketing authorisation. Therefore, when a ‘standard’/’full’ marketing authorisation has been already granted, it is not possible to subsequently change this authorisation into a conditional marketing authorisation or a marketing authorisation under exceptional circumstances.”
However, if a drug already has a conditional marketing authorization or has a marketing authorization under exceptional circumstances, EMA says it is possible to modify (including extend) the indication and related specific obligations.
Type-II variations: questions and answers
Extensions of marketing authorisations: questions and answers