Apel­lis coun­ters Alex­ion’s cri­tique, clears up FDA ques­tions as block­buster de­ci­sion looms

In Jan­u­ary, when Apel­lis re­vealed da­ta that showed their drug pegc­eta­coplan out­per­formed Alex­ion’s com­pa­ny-build­ing block­buster Soliris in treat­ing a rare blood dis­or­der called PNH, Alex­ion point­ed in­quir­ing re­porters to an en­zyme called LDH.

LDH ap­pears in near­ly all liv­ing cells and is par­tic­u­lar­ly com­mon in red blood cells. When those blood cells ex­plode — as hap­pens at dan­ger­ous rates in pa­tients with PNH, or parox­ys­mal noc­tur­nal he­mo­glo­bin­uria — the crum­bling cells re­lease LDH in­to the blood­stream. The pri­ma­ry end­point for Apel­lis’ piv­otal tri­al was he­mo­glo­bin lev­els, be­cause low­ered he­mo­glo­bin leads to many of the symp­toms. But you can al­so use LDH in a per­son’s blood to mea­sure how well a PNH drug is work­ing, if it’s pre­vent­ing the blood cells from dis­in­te­grat­ing.

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