Challenge

One of the key areas of EUDRAC’s work is the regulatory support of Orphan Drugs for rare diseases.  We are Sponsors for Orphan Drug Designations and have supported a successful PRIME application for a treatment for X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). This is an orphan disease which most significantly affects new-born males.  Babies are born with absent or abnormal ectodermal structures including a lack of sweat glands. The condition is associated with significant morbidities and an increased risk of infant mortality.

The PRIME scheme

The EMA PRIME scheme (PRIority Medicines) was launched two years ago in March 2016  to facilitate increased regulatory support for medicines being developed for unmet medical needs.  The aim of PRIME is to improve the availability of new medicines for patients for whom treatments currently either do not exist, or are inadequate .

Essentially, the EMA provides support at a much earlier stage in the development of a PRIME medicinal product and aims to be proactive in offering advice on development, particularly clinical trial design, to ensure that the authorisation of these medicines is as efficient as possible; it is expected that most PRIME medicines will be granted accelerated assessment by the CHMP.

PRIME application

A formal application for PRIME eligibility has to be made to the EMA. To be accepted, the applicant has to demonstrate that the medicine has a clear potential to provide beneficial treatment to patients with an unmet medical need; early clinical data must, therefore, provide sufficient evidence of efficacy.

Since its launch, there have been 177 applications for PRIME eligibility and 36 have been accepted (21%) including the application made by EspeRare for ER004, a protein replacement therapy for XLHED. EUDRAC provided EspeRare with regulatory support for this PRIME application and eligibility to PRIME was granted  for ER004 on 13 October 2017 based on the seriousness of the disease and the recognition that it represents an unmet medical need for which there is a lack of available treatment.

Approach

Kick-off meeting

EUDRAC participated in the pre-meeting telecon with the EMA in order to clearly identify who would be present at the Kick-off meeting and what information needed to be provide by EspeRare beforehand.  The EMA also explained the purpose of the meeting and what the specific aims would be with respect to EspeRare's project.

EUDRAC then reviewed the information package and presentation that was prepared by EspeRare before it was submitted to the EMA in advance of this Kick-off meeting. EUDRAC also attended the PRIME Kick-off meeting held at the EMA, together with the representatives from EspeRare.  The meeting discussed the future development plan for ER004, particularly the planned application for Protocol Assistance, and the support that will be provided by the EMA via its Committees and Expert Working Parties.

Protocol Assistance and future regulatory support

Following the outcome of the Kick-off meeting, EUDRAC has since participated in the pre-submission meeting between EspeRare and the EMA  in preparation for Protocol Assistance, and is now providing regulatory input into the Briefing Package and the specific questions to be included. 

Conclusion

This valuable experience of the PRIME scheme and the insight into how best to optimise communication with the EMA will be utilised for the future support of EspeRare's Orphan Drug Development for XLHED and other ODD that we are working on.