Un­en­cum­bered by safe­ty con­cerns, Roche's Hem­li­bra gets break­through sta­tus for new pa­tient group

Re­cent safe­ty con­cerns have not stopped Roche from ob­tain­ing a speedy FDA re­view for us­ing Hem­li­bra to treat he­mo­phil­ia A pa­tients with­out fac­tor VI­II in­hibitors, a mar­ket seg­ment cru­cial to Roche’s block­buster quest for its new ther­a­py ap­proved last No­vem­ber.

Roche rat­tled the mar­ket when it re­port­ed five deaths among pa­tients tak­ing Hem­li­bra, in­clud­ing two that emerged ear­li­er this year (The com­pa­ny em­pha­sized that all five were un­re­lat­ed to the drug). Jef­feries an­a­lyst David Stein­berg point­ed out that height­ened con­cerns could im­pact use in “non-in­hibitor pa­tients who are gen­er­al­ly well con­trolled” on Ad­vate, a fac­tor re­place­ment ther­a­py from chief ri­val Shire.

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