Al­ny­lam preps for an­oth­er PhI­II RNAi study, ‘break­through’ sta­tus in hand

Tout­ing ear­ly-stage ev­i­dence that its RNAi drug for rare cas­es of acute he­pat­ic por­phyr­ia had be­gun to demon­strate its promise in the clin­ic, Al­ny­lam to­day picked up the FDA’s break­through des­ig­na­tion for givosir­an, for­mer­ly known as ALN-AS1. And the biotech is plan­ning to use that in­side reg­u­la­to­ry track to hus­tle along a piv­otal study that will launch in a mat­ter of months.

The promi­nent Cam­bridge, MA-based biotech, which has had plen­ty of ups and downs over the years, says it plans to roll out some of the Phase I da­ta it’s been gath­er­ing on givosir­an in late June. Un­til then, the com­pa­ny says that reg­u­la­tors re­lied on ear­ly ev­i­dence on the “mean­ing­ful re­duc­tions in the oc­cur­rence of por­phyr­ia at­tacks” to pro­vide the BTD ti­tle. Late last year the biotech not­ed that re­sults” demon­strat­ed a ro­bust and durable low­er­ing of aminole­vulin­ic acid and por­pho­bilino­gen, the tox­ic heme in­ter­me­di­ates that are be­lieved to me­di­ate por­phyr­ia symp­toms and acute at­tacks.”

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