Decades in the mak­ing, Ky­owa Hakko Kirin fi­nal­ly wins FDA ap­proval for add-on Parkin­son's ther­a­py

More than two decades af­ter Ky­owa Hakko Kirin kicked off the clin­i­cal eval­u­a­tion of its Parkin­son’s dis­ease drug, the Japan­ese phar­ma group has fi­nal­ly se­cured FDA ap­proval.

The drug, istrade­fylline, tar­gets the adeno­sine A2A re­cep­tor, which is lo­cat­ed in the basal gan­glia — un­der­stood to play a key role in con­trol­ling vol­un­tary move­ment. It was ap­proved by the FDA as an add-on treat­ment to lev­odopa/car­bidopa on Tues­day. The drug was cleared for use in Japan back in 2013.

The drug, brand­ed as Nouri­anz, was sanc­tioned to treat Parkin­son’s pa­tients on the ba­sis of four 12-week place­bo-con­trolled clin­i­cal stud­ies that in­clud­ed a to­tal of 1,143 par­tic­i­pants. In each tri­al, pa­tients giv­en Nouri­anz ex­pe­ri­enced a sta­tis­ti­cal­ly sig­nif­i­cant de­crease from base­line in dai­ly ‘off’ time com­pared to those on a place­bo, the FDA said.

How­ev­er, its path to the fin­ish line with the FDA was not smooth sail­ing — Ky­owa Hakko Kirin had orig­i­nal­ly ap­plied for ap­proval more than a decade pri­or and was hand­ed a re­jec­tion by the agency in 2008. But the Japan­ese group elect­ed to plow ahead with its de­vel­op­ment pro­gram, al­though da­ta was not al­ways pris­tine. For ex­am­ple, in late 2016, a 12-week Phase III tri­al test­ed two dos­es of the drug against a place­bo, show­ing a trend to­wards ef­fi­ca­cy in Parkin­son’s pa­tients — but the re­sults were not sta­tis­ti­cal­ly sig­nif­i­cant.

The class of drugs Nouri­anz be­longs to has a tur­bu­lent past. In 2013, late-stage da­ta on Mer­ck’s adeno­sine A2A re­cep­tor ag­o­nist, preladenant, sug­gest­ed the drug was no bet­ter than a place­bo — which caused the US drug­mak­er to walk away from the mol­e­cule. Acor­da spent $363 mil­lion to swal­low Bi­otie, and its adeno­sine A2A re­cep­tor an­tag­o­nist — tozadenant — in 2016, but de­cid­ed to shelve its de­vel­op­ment the year af­ter, in re­sponse to un­sa­vory safe­ty da­ta that blight­ed a late-stage tri­al. Late last year, how­ev­er, Acor­da fi­nal­ly scored ap­proval for its in­haled lev­odopa ther­a­py, In­bri­ja.

The first line of ther­a­peu­tic de­fense for Parkin­son’s dis­ease pa­tients is typ­i­cal­ly lev­odopa/car­bidopa. How­ev­er, the ef­fec­tive­ness of oral lev­odopa is lim­it­ed by its short half-life — it re­mains in plas­ma for on­ly up to four hours fol­low­ing a sin­gle dose, re­quir­ing pa­tients to take mul­ti­ple dos­es dai­ly to fight ‘off’ pe­ri­ods of de­cline in mo­tor and non-mo­tor func­tion. Ex­ces­sive/in­ter­mit­tent oral dos­es of lev­odopa of­ten lead to in­vol­un­tary move­ments, or dysk­i­ne­sia, in some pa­tients.

Near­ly one mil­lion Amer­i­cans will be liv­ing with Parkin­son’s by 2020 — more than the com­bined num­ber of peo­ple di­ag­nosed with mul­ti­ple scle­ro­sis, mus­cu­lar dy­s­tro­phy and Lou Gehrig’s dis­ease, es­ti­mates the non-prof­it Parkin­son’s Foun­da­tion.

So­cial im­age: Ky­owa Hakko Kirin

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