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EMA orphan designation for medicines for rare diseases

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Today, for the Rare Disease Day, from Revio we would like to share the following information regarding the European Medicines Agency (EMA) orphan designation procedure for medicines for rare diseases.

An orphan medicine is a medicine used for the diagnosis, prevention, or treatment of a serious and rare and disease. To grant such designation, the developer of the medicine will have to fill an application that will be reviewed by the EMA’s Committee for Orphan Medicinal Products (COMP), meeting the following criteria:

  • Medicine must be intended to treat, prevent, or diagnose a life-threatening or chronically debilitating
  • The prevalence of the condition in the EU must not be more than 5 in 10.000.
  • No satisfactory method of diagnosis, prevention or treatment of the condition is authorised, or the medicine must be of significant benefit to those affected by the condition.


Sponsor of designated orphan medicines can benefit from a range of incentives offered in the European Union:

Protocol assistance

Protocol assistance is a form of scientific advice specifically for orphan medicines that is available at a reduced charge depending on the status of the sponsor. This procedure allows sponsors to get answers to their question on the types of studies needed to demonstrate the medicine’s benefits and risks and information on the significant benefit.

There is no restriction on the number of times a sponsor can request protocol assistance.

Access to the centralised authorisation procedure

All designated orphan medicines are assessed for marketing authorisation centrally in the European Union. This allows companies to make a single application to the EMA, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States.

Ten years of market exclusivity

Authorised orphan medicines benefit from ten years of protection from market competition with similar indications once they are approved. This period of protection is extended by two years for medicines that also have complied with an agreed paediatric investigation plan granted at the time of review of the orphan medicine designation.

Additional incentives for small and medium-sized enterprises

SMEs benefit from further incentives when developing medicines with orphan designation. These include administrative and procedural assistance from the Agency’s SME office and fee reduction (e.g., 100% fee reduction for protocol assistance).

Fee reductions

Companies applying for designated orphan medicines pay reduced fees for regulatory activities such as protocol assistance, marketing authorisation applications, inspections before authorisation, applications for changes to marketing authorisations made after approval, and reduced annual fees.

Orphan Advanced therapies for medicines for rare diseases 

Many Advanced Therapy Medicinal Products (ATMPs) have been granted Orphan designation before their approval in the last years. In this table are summarized the advanced therapies that were granted Orphan designation in its development, the rare disease intended to treat, the classification and date of approval. Click the name to know more about EMA orphan designation for medicines for rare diseases.

HoloclarTissue engineered productReplacement of damaged cells on the corneaFeb 2015
Strimvelis Gene therapy productSevere combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)May 2016
KymriahGene therapy productB-cell acute lymphoblastic leukemia (ALL) and diffuse large B-cell lymphoma (DLBCL)Aug 2018
LuxturnaGene therapy productLoss of vision due to retinal dystrophyNov 2018
YescartaGene therapy productDiffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL)May 2018
ZolgensmaGene therapy productSpinal muscular atrophyMay 2020
ZyntegloGene therapy productBeta thalassemiaMay 2019
AlofiselSomatic cell therapy productComplex anal fistulas associated with Crohn’s diseaseMar 2018
SkysonaGene therapy productEarly cerebral adrenoleukodystrophy (CALD)Jul 2021
AbecmaGene therapy productMultiple myelomaAug 2021


Cell therapy productMantle cell lymphomaDec 2020


Cell therapy productDiffuse large B cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B)Jan 2022


We hope you find this summary of orphan designation for medicines for rare diseases useful and of interest. If you would like to discuss any of these updates with the team at REVIO, please get in touch here.

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