An FDA re­jec­tion let­ter for Cor­cept Ther­a­peu­tics’ po­ten­tial hor­mon­al dis­or­der drug says the agency told the com­pa­ny it had se­ri­ous con­cerns be­fore the ap­pli­ca­tion was sub­mit­ted.

The com­plete re­sponse let­ter, re­leased Fri­day, again shows how some com­pa­nies have not ful­ly ex­plained the rea­sons be­hind a re­jec­tion de­ci­sion in their pub­lic an­nounce­ments. This is the first CRL the FDA has re­leased in 2026, fol­low­ing the agen­cy's first-ever re­lease of more than 200 re­jec­tion let­ters last year.

On Dec. 31, 2025, Cor­cept an­nounced the FDA's re­jec­tion of rela­co­ri­lant as a drug for hy­per­ten­sion sec­ondary to Cush­ing's syn­drome in a re­lease, not­ing the FDA "could not ar­rive at a fa­vor­able ben­e­fit-risk as­sess­ment for rela­co­ri­lant." At the time, the CEO said the com­pa­ny was "sur­prised" by the re­jec­tion.

EU reg­u­la­tors have re­fused to green­light Eli Lil­ly’s weight loss drug Moun­jaro as a treat­ment for a type of heart fail­ure, while giv­ing the go-ahead for No­vo Nordisk’s semaglu­tide in liv­er dis­ease.

Lil­ly failed to get sup­port from the Eu­ro­pean Med­i­cines Agency’s hu­man med­i­cines com­mit­tee, or CHMP, on Fri­day for Moun­jaro to treat chron­ic heart fail­ure with pre­served ejec­tion frac­tion in adults with obe­si­ty. HF­pEF is when the heart be­comes too stiff to pump enough blood around the body.

The com­mit­tee said Moun­jaro did­n't re­duce the num­ber of pa­tient deaths due to heart prob­lems in pa­tients with obe­si­ty and HF­pEF. While the drug did re­duce the num­ber of heart fail­ure-re­lat­ed hos­pi­tal­iza­tions, it's un­clear whether that was due to the weight loss-in­de­pen­dent ef­fect of Moun­jaro, CHMP said.

Ul­tragenyx has re­sub­mit­ted its ap­proval re­quest for a rare dis­ease gene ther­a­py af­ter the FDA re­ject­ed it last sum­mer.

UX111 is an AAV gene ther­a­py de­signed to treat San­fil­ip­po syn­drome type A. The com­pa­ny is again seek­ing ac­cel­er­at­ed ap­proval us­ing a nov­el bio­mark­er, part of a push by bio­phar­ma to test the FDA’s ap­petite to ease ac­cess for rare dis­ease drugs.

But in Ju­ly, the agency turned down the drug af­ter an in­spec­tion of a man­u­fac­tur­ing fa­cil­i­ty raised con­cerns. Ul­tragenyx said Fri­day that its re­sub­mis­sion in­cludes “com­pre­hen­sive” re­spons­es to those con­cerns, in ad­di­tion to longer-term da­ta from pa­tients. If the agency ac­cepts the re­sub­mis­sion, Ul­tragenyx said the de­ci­sion could come in the third quar­ter of this year.

Bit­ter­root Bio, a biotech from lead­ing minds in the CD47 field, ap­pears to have re­turned to the dis­cov­ery stage af­ter at­tempt­ing to ex­pand the po­ten­tial of the "don't eat me" sig­nal from the can­cer field in­to the car­dio­vas­cu­lar realm.

The Pa­lo Al­to-based biotech had shipped a CD47-tar­get­ing im­munomod­u­lat­ing pro­tein drug in­to a Phase 2a study in Aus­tralia. The tri­al of BRB-002 be­gan dos­ing pa­tients with ath­er­o­scle­ro­sis in June 2025. That work ap­pears to have hit a snag, send­ing Bit­ter­root back to the draw­ing board.

De­part­ing CEO Pa­van Cheru­vu said in a LinkedIn post Thurs­day that Bit­ter­root will re­struc­ture to "fo­cus on dis­cov­ery bi­ol­o­gy and trans­la­tion­al re­search" and will "lever­age nov­el bi­o­log­i­cal in­sights from the tri­al that cre­ate unique op­por­tu­ni­ties for Bit­ter­root to ad­vance the sci­ence of the CD47 field."