Kids with SMA to re­ceive costly drug on com­pas­sion­ate grounds

Jerusalem Post - - NEWS - • By JUDY SIEGEL

Up to 15 chil­dren with se­vere cases of de­bil­i­tat­ing spinal mus­cu­lar at­ro­phy will re­ceive the drug Spin­raza, which costs NIS 2 mil­lion a year per pa­tient.

The or­phan drug will be sup­plied by its de­vel­oper, Bio­gen Inc., for com­pas­sion­ate rea­sons, Health Min­is­ter Ya’acov Litz­man told the Fi­nance Com­mit­tee on Tues­day.

A to­tal of 80 Is­raeli young­sters suf­fer from the rare ge­netic dis­ease, which can sig­nif­i­cantly im­prove with the help of the drug. The Euro­pean Com­mis­sion ap­proved the drug for mar­ket­ing six months af­ter the FDA ap­proved it to treat chil­dren with SMA. Litz­man has in­structed all hos­pi­tals to pro­vide the drug as an im­me­di­ate re­sponse to SMA for all the se­ri­ous (type 1) pa­tients suf­fer­ing from the dis­ease.

SMA is a rare neu­ro­mus­cu­lar dis­or­der that causes pro­gres­sive mus­cle wast­ing, which of­ten leads to early death. Spin­raza (nusin­ersen) has been found to sig­nif­i­cantly im­prove the pa­tient’s func­tion­ing. The min­is­ter told the com­mit­tee that he is work­ing on a way to pro­vide treat­ment to ev­ery pa­tient who needs the drug, but did not ex­plain how.

It the drug had to be sup­plied by the bas­ket of health ser­vices for 2018, whose con­tents are be­ing de­cided on now, al­most one third would have gone to pay for Spin­raza. The health bas­ket com­mit­tee learned that Bri­tain faces a sim­i­lar dilemma and will sup­ply the drug to some of its af­fected chil­dren.

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