EMA changes orphan designation policy for certain eye drugs to expand access
The European Medicines Agency said yesterday that it’s changing how it signs off on orphan designations for drugs targeting inherited retinal dystrophies (IRD) in order to make sure that patients are not left out because of the limitations of the previous orphan designation terms.
Inherited retinal dystrophies are a group of genetic and progressive blinding diseases.
The Committee for Orphan Medicinal Products for the EMA announced the changed policy, which the organization said was supported by input from experts and patients of IRD, as well as a review of scientific literature and 64 active orphan designations in the specific eye diseases in the EU.
“Based on a thorough review, supported by a consultation of IRD clinical experts and patients, the COMP has adopted a new approach for designating conditions in IRDs,” the statement reads.
There are now three options for companies wanting to submit an orphan drug application into four different groups: non-syndromic, syndromic, inherited choroidal dystrophies and hereditary vitreoretinopathies.
If a therapy is “relatively broadly applicable” in IRD, terms for the application can be selected from any of the four new groups. But if a drug that can be broadly used can target more than one of the four groups, multiple orphan designations could be necessary.
For gene therapies developed for eye diseases, the condition in the orphan drug designation application has to be built from the term “inherited retinal dystrophy due to dysfunction in the target-gene.”
If a drug doesn’t fit into any of the four groups, an “occasional singular orphan designation” may still be necessary for a non-gene therapy drug.
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.
“Using classically derived IRD names for the orphan condition may mean that some otherwise treatable patients may be out of scope of an approved treatment if these patients show different signs and symptoms to the classical group,” the explanation from the committee reads.
From now on, the committee says that any companies submitting orphan drug designation applications “specify the orphan condition applied for and fully justify the chosen approach.” Any companies with existing orphan designations should consider making any changes in line with the new groups before submitting a marketing authorization application.