Monica Gomar Mengod x

It is not possible to submit an initial protocol assistance request if the transfer of the designation for orphan medicinal product has not been finalised yet. On receipt of the EC decision, EMA will complete the case, the ODD Regulatory Entitlement will be transferred, and thereafter the applicant will be able to proceed and apply for protocol assistance.

Annual reports for orphan medicinal products designated by the EU should be submitted within 2 months following the anniversary of the grant of the designation, meaning the EC decision.

 

We certainly welcome submission of annual reports of orphan medicinal products designations in parallel if it helps to streamline the process at your end. 

After checking BioMarin records, this enquiry has turned up to be too specific to answer via this channel. The EMA Orphan Office is currently in direct discussion with sponsor outside this portal to fix the contacts and associated access roles for their organisation. 

The fields on 'compassionate use', 'marketing authorisation' and 'new orphan designation' relate to global development (not only EU). Please mention any marketing authorisations (in the relevant condition/indication) obtained anywhere in the world.

Unfortunately only the current sponsor of an orphan medicinal product designation can trigger a transfer of sponsorship.  As it appears that the current sponsor did not inform EMA and EC before it ceased to exist, there is no other option than for the new sponsor to apply for the orphan designation again. Please kindly let us know the orphan designation you are referring to, for us to put a note for the record and inform the EC. 
 

As you might be aware the rule is that annual reports for orphan medicinal products designated by the EU should be submitted within 2 months following the anniversary of the grant of the designation or at World Rare Disease Day (last day of February). Nevertheless, your proposal is acceptable.
 

Since the implementation of the IRIS online portal for all submission related to orphan procedures, joint submissions to EMA/FDA are no longer possible, including for annual reports. 

Annual reports for orphan medicinal products designated by the EU should be submitted via the IRIS online portal and within 2 months following the anniversary of the grant of the designation or at World Rare Disease Day (last day of February).

https://www.ema.europa.eu/en/human-regulatory/research-development/orphan-designation/submitting-annual-reports-medicine-development
 

Thank you for your question.
Annual reports for orphan medicinal products designated by the EU should be submitted within 2 months following the anniversary of the grant of the designation or at World Rare Disease Day (last day of February).

Monica Gomar Mengod
Orphan Medicines Office