EMA Committee Announces Orphan Drug Application Changes to Improve Access to Rare Eye Disease Medications

Policies regarding orphan drug designations for a group of rare genetic eye diseases called inherited retinal dystrophies are changing, according to the European Medicines Agency (EMA). The changes would target terminology regarding the categories of disorders that are covered under the orphan drug designation, allowing drugs to be submitted into one or more of four subgroups.

According to Katherine Lewin, “But why the change? The committee decided that IRDs are far too complex to continue to use traditional terms that were developed when the genetics behind the diseases weren’t understood and were solely based on symptoms.”

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(Source: Endpoints News, January 25^th, 2023)