Al­ny­lam shoots for a speedy FDA OK af­ter their next shot at field­ing a pricey rare dis­ease drug hits PhI­II goal

Al­ny­lam $AL­NY is go­ing for it.

Af­ter rack­ing up a pos­i­tive set of da­ta for an in­ter­im re­view of their piv­otal study for givosir­an, ex­ecs at the RNAi pi­o­neer say they’ll hus­tle ahead with their sec­ond shot at a new drug ap­proval for a rare dis­ease, just weeks af­ter gain­ing a his­toric green light for their lead drug patisir­an.

We’re not get­ting any hard da­ta in the state­ment out Thurs­day morn­ing, just con­fir­ma­tion that re­searchers hit their marks on sta­tis­ti­cal sig­nif­i­cance in an in­ter­im look at a key bio­mark­er. And that leaves them on track to gain a po­ten­tial ac­cel­er­at­ed ap­proval, es­pe­cial­ly as new man­age­ment at the FDA is quick­er than ever in push­ing out new drugs for rare dis­eases and can­cer ahead of fi­nal ef­fi­ca­cy and safe­ty da­ta.

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