An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tu­mul­tuous ride for Or­p­hazyme con­tin­ued on Fri­day as the com­pa­ny an­nounced that a piv­otal tri­al for its lead drug ari­mo­clo­mol failed yet again, this time in the treat­ment of ALS, seed­ing doubt in a drug that had re­cent­ly been cleared by the FDA for pri­or­i­ty re­view. The lat­est fail­ure casts a dark­er shad­ow on the up­com­ing de­ci­sion de­spite Or­p­hazyme’s up­beat out­look.

In a state­ment, the Dan­ish biotech an­nounced that the drug did not meet its pri­ma­ry or sec­ondary end­points eval­u­at­ing func­tion and sur­vival. But the com­pa­ny has not an­nounced any da­ta sur­round­ing the fail­ure, in­stead say­ing that it will pub­lish the com­plete re­sults lat­er this year.

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