EMA Orphan Drug Designation Consulting

Securing EMA Orphan Drug Designation (ODD) is a critical milestone for rare disease therapeutics. Get EMA ODD assistance from the experts at Only Orphans Cote today.

Trusted Authority in FDA & EMA Orphan Drug Designation

98% First-Attempt EMA Application Success Rate

Expert Guidance from the Former FDA Director of Orphan Products

What is EMA Orphan Drug Designation?

Only Orphans Cote is the global authority in Orphan Drug Designation (ODD) applications. With unparalleled knowledge of the FDA Office of Orphan Products Development (OOPD) process and its unwritten rules, we achieve close to a 95% first-attempt approval rate compared to an industry average of just 35%.

The European Medicines Agency (EMA), through the Committee for Orphan Medicinal Products (COMP), grants orphan designation to drugs targeting life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the EU.


This designation is an economic entitlement that provides long-term commercial benefits and strengthens the development pathway.

Typical EMA Designation Timeline

  • Application review without pre-submission meeting: ~8 months
  • Application review with pre-submission meeting: ~10 months

Our Comprehensive EMA ODD Support

Our clients have secured EMA ODD across multiple therapeutic areas, enabling access to critical regulatory incentives and solidifying their long-term rare disease development strategies.


Common EMA Challenges & Our Solutions:


Navigating Complex EU Prevalence Requirements

We guide sponsors in preparing robust epidemiology data.


Expressing Significant Benefit Demonstration

We help articulate therapeutic advantage over existing treatments.


EMA Annual Reporting

We manage yearly submissions, ensuring ongoing compliance.


Orphan drug consultant and physician sitting together

EMA Eligibility & Requirements


  1. Life-threatening or chronically debilitating condition:  The medicine must be intended for a condition that is serious, rare, and has a significant impact on patients.
  2. Prevalence threshold: The condition must not affect more than 5 in 10,000 people in the European Union at the time of application, or the product must be unlikely to generate sufficient return on investment without incentives.
  3. No satisfactory treatment available: Either no approved treatment method exists, or the medicine will provide a significant benefit over existing therapies (e.g., improved efficacy, safety, or major contribution to patient care).
  4. Medical plausibility: Preclinical or clinical data must show a reasonable basis that the medicine could diagnose, prevent, or treat the condition.
INDs

Ready to Secure EMA Orphan Drug Designation?

We offer a free initial data review to evaluate EMA orphan designation readiness. Our team will advise whether your program qualifies before you invest in a full application. Then, we’ll design the clearest path to help your program win EMA designation.