On November 18, 2016, EMA published an update to the post-authorisation guidance on MA extensions and type II variations to include further details on the information companies need to provide when submitting a Type II variation or a MA extension for an orphan-designated medicine.
This follows the publication of a new Notice by the EC on the application of Articles 3, 5 and 7 of Regulation (EC) No 141/2000 on OMPs, which provides guidance on orphan designation provisions. One of the key changes introduced by the Notice is the requirement to fulfill the OD criteria for any new therapeutic indication falling under a previously confirmed Orphan Designation, meaning that the COMP will have to consider whether the specific scope of the variation is appropriately justified in the context of the OD criteria. This aims at ensuring that the orphan MA only covers indications that fulfill the criteria foreseen in the Orphan Regulation.
The main updates relate to a product that has been designated as an orphan when the application concerns a new therapeutic indication or a modification of an existing one, a COMP review may be required as follows:
- for a new therapeutic indication falling within a new orphan designation, the COMP will have to confirm the maintenance of the OD before authorisation of the new indication. In this case, the sponsor should provide at the time of submission a maintenance report;
- for a new therapeutic indication falling within an already authorised orphan designation, the COMP will have to consider if the specific scope of the variation raises justified and serious doubts in respect to the fulfilment of the orphan designation criteria and indicate if a formal review process of the maintenance of the OD is needed. In this case, the sponsor should provide upon submission either a justification that the variation does not raise doubts on the fulfilment of the orphan criteria or a maintenance report to justify that the orphan criteria are still met.
As of 18 November 2016, EMA’s post-authorisation guidance should be followed for all newly submitted applications. Marketing authorisation holders are advised to contact the Agency in advance of their upcoming orphan extension of indication for further advice.