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.

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