Biogen reverses course on compassionate use for ALS drug tofersen with plans to open program in July

Josh Sullivan

Associate Editor

De­spite im­mense pres­sure from the ALS com­mu­ni­ty, Bio­gen had re­peat­ed­ly re­fused to open in­ves­ti­ga­tion­al drug tofersen for use out­side of clin­i­cal tri­als. But now, cav­ing to that de­mand, Bio­gen has re­versed its stance.

With a piv­otal Phase III study in ALS on­go­ing and da­ta ex­pect­ed to read out lat­er this year, Bio­gen just a month ago said it wouldn’t of­fer tofersen for com­pas­sion­ate use in an at­tempt to be fair to pa­tients on place­bo in that late-stage test. The orig­i­nal goal was to wait for top-line ef­fi­ca­cy and then open an Ear­ly Ac­cess Pro­gram for pa­tients with a spe­cif­ic mu­tant vari­a­tion of the SOD1 gene that comes with a par­tic­u­lar­ly poor prog­no­sis.

But now, fac­ing more than 70,000 pe­ti­tion­ers as of March, Bio­gen will open a com­pas­sion­ate-use pro­gram start­ing in mid-Ju­ly, once the study clos­es but be­fore the first ef­fi­ca­cy read­outs. In a state­ment, Bio­gen said it still be­lieved it was un­fair to of­fer the drug ear­ly be­fore pa­tients on place­bo were giv­en a chance to take it, but the drug­mak­er had soft­ened its stance on wait­ing for the fall:

We do not be­lieve it is fair to ask par­tic­i­pants in this study to con­tin­ue to re­ceive place­bo while oth­er SOD1-ALS pa­tients are of­fered ac­cess to tofersen, but we do be­lieve that ac­cess could be pro­vid­ed as soon as the place­bo-con­trolled study has end­ed. How­ev­er, un­til the safe­ty and ef­fi­ca­cy have been es­tab­lished, we will pri­or­i­tize ear­ly ac­cess for a sub­set of the most rapid­ly pro­gress­ing pa­tients with this rare, se­vere dis­ease.

The pro­gram will en­roll pa­tients with the most dire form of SOD1-mu­tant ALS with el­i­gi­bil­i­ty cri­te­ria de­vel­oped along­side a board of in­de­pen­dent ex­perts and bioethi­cists, Bio­gen said. The drug will be re­served for pa­tients with an ALS­FRS-R slope de­cline greater than or equal to 2 points per month.

The about-face comes as a big win for pa­tients and ad­vo­cates spurred on by health­care com­mu­ni­ca­tions ex­ec­u­tive and SOD1-ALS pa­tient Lisa Stock­man Mau­riel­lo, Bloomberg re­port­ed.

Tofersen’s Phase III study is set to be com­plet­ed on Aug. 31, and Bio­gen still plans to open an ear­ly ac­cess pro­gram as it moves through the po­ten­tial reg­u­la­to­ry ap­proval process. The im­me­di­a­cy for pa­tients like Mau­riel­lo comes from the AD5 vari­ant of SOD1-ALS, which of­ten kills pa­tients with­in 12 months from their first symp­toms.