Bio­Marin an­nounced Mon­day af­ter­noon that it's ex­plor­ing ways to di­vest Roc­ta­vian from its port­fo­lio, fol­low­ing a rocky two years since the he­mo­phil­ia A gene ther­a­py was first ap­proved.

The move is­n't sur­pris­ing since up­take of the gene ther­a­py has been ex­treme­ly lim­it­ed, and the com­pa­ny has pre­vi­ous­ly raised the ques­tion of di­vest­ing Roc­ta­vian. The ther­a­py has gen­er­at­ed $23 mil­lion in rev­enue so far this year, in­clud­ing just $3 mil­lion in the third quar­ter.

Roc­ta­vian’s strug­gles to find its foot­ing in the mar­ket are an­oth­er ex­am­ple of the com­mer­cial chal­lenges faced by de­vel­op­ers of gene ther­a­pies. Oth­er high-pro­file com­pa­nies such as blue­bird bio have sim­i­lar­ly faced a tough time gen­er­at­ing rev­enue for their gene ther­a­pies. Once a force in gene ther­a­py, blue­bird was bought by a pair of in­vestors for on­ly $29 mil­lion up­front ear­li­er this year as the com­pa­ny ran out of op­tions and mon­ey.

The UK is giv­ing phar­ma com­pa­nies more time to de­cide whether to leave the coun­try’s drug re­bate scheme as ten­sion con­tin­ues over the gov­ern­ment’s in­vest­ment in the sec­tor.

The UK gov­ern­ment and trade group the As­so­ci­a­tion of British Phar­ma­ceu­ti­cal In­dus­try ex­tend­ed the dead­line by two weeks for com­pa­nies to ex­it the UK's Vol­un­tary Scheme for Brand­ed Med­i­cines Pric­ing and Ac­cess (VPAG), ac­cord­ing to a Tues­day re­lease. VPAG is the per­cent­age of sales a com­pa­ny pays back to the UK's Na­tion­al Health Ser­vice.

VPAG launched last year in col­lab­o­ra­tion with the gov­ern­ment and in­dus­try and was de­signed to low­er the cost the NHS pays for brand­ed drugs. Drug­mak­ers have been lob­by­ing for the gov­ern­ment to low­er its VPAG rate, which is set at near­ly 23%. But the rate re­mains the same af­ter UK Health Sec­re­tary Wes Street­ing failed to strike a drug-pric­ing deal with phar­ma com­pa­nies in Au­gust.

Plus, news about Cy­bin, In­cyte, Roche, Mer­ck, Ei­sai, Ne­upho­ria Ther­a­peu­tics, Aldeyra Ther­a­peu­tics and X4 Phar­ma­ceu­ti­cals:

🏭 FDA is­sues CRL to Re­gen­eron: The drug­mak­er has of­fi­cial­ly been hand­ed a com­plete re­sponse let­ter for its pre-filled sy­ringe for Eylea HD. The com­pa­ny had warned in Au­gust that it ex­pect­ed reg­u­la­to­ry de­lays af­ter is­sues were found at a Catal­ent site in Bloom­ing­ton, IN, that’s now owned by No­vo Nordisk. The plant has since been is­sued an “of­fi­cial ac­tion in­di­cat­ed” clas­si­fi­ca­tion. An­oth­er cus­tomer of the In­di­ana site, Schol­ar Rock, al­so re­ceived a CRL re­lat­ed to the plant. — An­na Brown

GSK said Tues­day it will spend $85 mil­lion up­front for an ex­clu­sive li­cense to a Phase 1-stage small in­ter­fer­ing RNA (siR­NA) prod­uct be­ing de­vel­oped by Em­piri­co to treat COPD.

Em­piri­co will over­see the on­go­ing Phase 1 study of EMP-012, which a GSK spokesper­son said is due to read out next year. Af­ter that, GSK will take over clin­i­cal de­vel­op­ment, ap­proval fil­ings and sales world­wide.

The UK phar­ma could fork over up to $660 mil­lion more if EMP-012 meets de­vel­op­ment, reg­u­la­to­ry and com­mer­cial goals, plus tiered sales roy­al­ties.

This is the sec­ond deal an­nounced by GSK this week, which will re­port its third-quar­ter earn­ings Wednes­day. On Mon­day it took ex­clu­sive rights to a pre­clin­i­cal an­ti­body-drug con­ju­gate for prostate can­cer, promis­ing orig­i­na­tor Syn­di­via up to £268 mil­lion ($357 mil­lion), in­clud­ing the up­front and biobucks, with roy­al­ties on top.