Al­ny­lam flags loom­ing FDA ap­pli­ca­tion for givosir­an, adding an­oth­er round of stel­lar ef­fi­ca­cy da­ta from ear­ly study

Al­ny­lam $AL­NY is buff­ing up its da­ta on givosir­an in an­tic­i­pa­tion of launch­ing an as­sault on an ac­cel­er­at­ed ap­proval lat­er in the year.

The biotech is of­fer­ing up some long-term ex­ten­sion da­ta from a small, ear­ly-stage tri­al of its num­ber two RNAi drug in the pipeline. Re­searchers are stick­ing with the low dose of the drug af­ter track­ing a big drop in the rate of acute he­pat­ic por­phyr­ia at­tacks along with lev­els of aminole­vulin­ic acid, be­lieved to be the pri­ma­ry dis­ease trig­ger for the ul­tra-rare dis­ease, and the bio­mark­er for por­pho­bilino­gen.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER