FDA ad­vis­ers tout gene ther­apy as treat­ment for some blind­ness

Texarkana Gazette - - RECORDS/NEWS -

SIL­VER SPRING, Md.—A po­ten­tially ground­break­ing treat­ment for a rare form of blind­ness moved one step closer to U.S. ap­proval Thurs­day, as fed­eral health ad­vis­ers en­dorsed the ex­per­i­men­tal gene ther­apy for pa­tients with an in­her­ited con­di­tion that grad­u­ally de­stroys eye­sight.

The panel ex­perts to the Food and Drug Ad­min­is­tra­tion voted unan­i­mously in fa­vor of Spark Ther­a­peu­tics’ in­jectable ther­apy, which aims to im­prove vi­sion by re­plac­ing a de­fec­tive gene needed to process light.

The vote amounts to a rec­om­men­da­tion to ap­prove the ther­apy. The FDA has un­til mid-Jan­uary to make its de­ci­sion and does not have to fol­low the panel’s rec­om­men­da­tion, though it often does.

If ap­proved, Lux­turna would be the first gene ther­apy in the U.S. for an in­her­ited dis­ease and the first in which a cor­rec­tive gene is given di­rectly to pa­tients. While the ther­apy from Spark Ther­a­peu­tics tar­gets a small group of pa­tients— about 2,000 in the U.S.—ex­perts say it could pave the way for other ge­netic treat­ments for a va­ri­ety of in­her­ited con­di­tions.

Panelists de­bated sev­eral specifics of the treat­ment, split­ting on whether to re­quire a min­i­mum age for treat­ment; the com­pany says the drug is in­tended for those 3 years and older.

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