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.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”