On the same day that Glax­o­SmithK­line sought to as­sure in­vestors that the com­pa­ny’s new R&D team would de­liv­er im­por­tant drugs in the fu­ture, its re­searchers work­ing on Nu­cala (mepolizum­ab) were slapped down in their at­tempt to gain an added OK for COPD.

A pan­el of out­side ex­perts vot­ed 16 to 3 against a mar­ket­ing ap­proval. That isn’t bind­ing, but af­ter the in­ter­nal re­view­ers at the FDA of­fered a scathing re­view of their da­ta, it’s all but a done deal now — bar­ring some un­ex­pect­ed last minute in­ter­ven­tion in its fa­vor.

AD­COMM VOTE: Is the ben­e­fit-risk pro­file ad­e­quate to sup­port ap­proval of #mepolizum­ab as add-on treat­ment to in­haled cor­ti­cos­teroid-based main­te­nance treat­ment for the re­duc­tion of ex­ac­er­ba­tions in pa­tients with #COPD guid­ed by blood eosinophil counts? $GSK
3-Yes
16-No
0-Ab­stain

— SAC Track­er (@FDAad­comm) Ju­ly 25, 2018

The FDA was clear­ly unim­pressed with GSK’s pack­age.

GSK’s drug failed one late-stage test while scor­ing a suc­cess in the oth­er. With on­ly two Phase III stud­ies in this in­di­ca­tion, that left the pan­el to con­sid­er an ap­proval with­out sup­port­ing da­ta from two well-con­trolled stud­ies — the agency gold stan­dard.

There was no dose re­sponse they could find in the two dosage arms, which didn’t help.

The FDA added:

Some key sec­ondary end­points, such as time to first mod­er­ate-to-se­vere COPD ex­ac­er­ba­tion showed nu­mer­i­cal trends fa­vor­ing mepolizum­ab. How­ev­er, there were no con­sis­tent and clin­i­cal­ly mean­ing­ful dif­fer­ences in se­vere COPD ex­ac­er­ba­tion rate, FEV1, or SGRQ be­tween mepolizum­ab 100 mg and place­bo.

GSK’s tri­al de­sign al­so spurred ques­tions, with no base­line col­lec­tion of asth­ma his­to­ry — which could have in­flu­enced the out­come of the study. How will GSK prop­er­ly de­fine the pa­tient pop­u­la­tion for this drug?

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”