Aussies’ anx­ious wait for CF won­der drug

Central and North Burnett Times - - READ -

ADRUG that treats the un­der­ly­ing cause of cys­tic fi­bro­sis in some pa­tients is go­ing back to the Phar­ma­ceu­ti­cal Ben­e­fits Ad­vi­sory Com­mit­tee for the third time.

Clin­i­cal tri­als in Aus­tralia and across the world pro­vide ev­i­dence that Orkambi, a new gen­er­a­tion drug for cys­tic fi­bro­sis, im­proved lung func­tion and re­duced ex­ac­er­ba­tions, hos­pi­tal­i­sa­tion and an­tibi­otic use in trial par­tic­i­pants.

The drug is de­signed for those with the cys­tic fi­bro­sis gene mu­ta­tion F508del ho­mozy­gous.

“Aus­tralia is a coun­try that prides it­self on an eq­ui­table and fair health sys­tem but sadly the rel­e­vant par­ties, the govern­ment’s PBAC and the multi­na­tional phar­ma­ceu­ti­cal com­pany Ver­tex, have been un­able to come to an agree­ment on costs for this life-chang­ing drug,” the chief ex­ec­u­tive of Cys­tic Fi­bro­sis Aus­tralia Net­tie Burke said.

“We know ev­ery­one is do­ing ev­ery­thing they can at the ne­go­ti­at­ing table and the Fed­eral Health Min­is­ter Greg Hunt in par­tic­u­lar has been par­tic­u­larly sup­port­ive. But for the sake of so many peo­ple’s fu­ture we need to close that gap now.”

Where the cur­rent treat­ment ar­se­nal for cys­tic fi­bro­sis is lim­ited to therapies that treat the symp­toms and clin­i­cal man­i­fes­ta­tions of the dis­ease, Orkambi is de­signed to treat the un­der­ly­ing cause of CF.

The 96-week trial out­comes showed that lung dam­age was slowed on av­er­age by 40%.

Ex­ac­er­ba­tions re­duced by 39%, hos­pi­tal­i­sa­tions were re­duced by 61% and an­tibi­otic use by 56%.

Orkambi also had a pos­i­tive flow-on ef­fect on the men­tal health of peo­ple with CF lead­ing to so­cial in­clu­sion.

“The PBAC must re­alise that Aus­tralians with CF need ac­cess to life-chang­ing drugs,” Ms Burke said.

“Specif­i­cally and im­por­tantly in this in­stance, Orkambi is the first treat­ment that can pre­vent or at least slow down per­ma­nent, ir­re­versible lung dam­age for peo­ple with CF who have two copies of F508del gene mu­ta­tion.”

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