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Orphan Drug Designation

If you are developing a medicine for a rare disease, orphan drug designation can be critical to commercial success. We have extensive experience helping clients develop medicines for rare diseases.

How we can help

  • plan your orphan strategy,
  • write and submit your orphan drug designation,
  • support any regulatory interactions regarding your orphan application,
  • guide and support your orphan similarity report in the lead-up to your marketing authorization application (MAA) submission, and
  • plan your orphan maintenance strategy at the time of MAA submission.

A comprehensive orphan maintenance strategy is pivotal to retaining orphan designation and it is often advisable to take scientific advice on this aspect of the development strategy.


Download our free guide

This guide explains when to submit the orphan application and how to plan for the maintenance report submission at the time of the MAA.


What is orphan designation status?

Orphan designation status is granted to companies developing medicines for rare diseases. Being orphan designated means that a company has fulfilled the requirements set out by the European Medicines Agency (EMA) or the Food and Drug Administration (FDA) and can benefit from fee incentives and exclusivity protections.

What are the criteria for orphan designation in the European Union (EU)?

The EMA stipulates that small molecules/advanced therapies are developed for life-threatening illnesses; where there are no satisfactory existing methods of diagnosis, prevention or treatment; and, the prevalence in the EU isn’t greater than 5 in 100,000 people.

What are the benefits of orphan designation?

Companies developing orphan medicines for rare diseases can benefit from 10 years of market exclusivity, approval via the centralised procedure, research grants and fee reductions associated with regulatory activities.

years of regulatory expertise

clients supported

agency interactions

orphan applications

marketing applications

We have supported 30+ orphan applications