RARE Daily

EMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies

January 26, 2023

The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its policy on orphan designation for a set of genetic eye disorders known as inherited retinal dystrophies, a recognition of the complexity of these conditions.

The committee created three options for an orphan designation for inherited retinal dystrophies (IRDs). These include therapies that are relatively broadly applicable in IRDs, targeted gene therapies, and a singular orphan designation for non-gene therapy products.

The committee said the genetics of IRDs are complex. It is now understood that one abnormal gene can have different clinical appearances. It is also understood that one phenotype can be associated with variants in more than 60 different genes.

Previously, the committee used classical IRDs terms as orphan designation conditions, but with the expanded understanding of these conditions orphan designation in IRDs is no longer straightforward.

The committee in making its changes undertook a review to ascertain the state of the art in IRDs to assess what would be the best set of terms for orphan designation for IRD therapies. The review included a literature review that included an overview of 64 active orphan designations in IRDs in the European Union including the nature of the product, possible alternative grouping systems of IRDs, and a consultation of IRD clinical experts and patients.

Sponsors with existing orphan designations in IRDs may consider amending their designation before filing a marketing authorization application or protocol assistance if the existing designation would not cover the intended target patient population.

For new submissions, sponsors should specify the orphan condition applied for and fully justify the chosen approach in line with the recommendations in this document and the orphan legislation and guidance as these will be considered by the COMP when deciding on the OD application.

Author: Rare Daily Staff

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