For the sec­ond straight day, the FDA’s Cel­lu­lar, Tis­sue and Gene Ther­a­pies ad­comm vot­ed unan­i­mous­ly in fa­vor of FDA ap­prov­ing a blue­bird bio gene ther­a­py, this time by a 13-0 vote in fa­vor of beti-cel as a po­ten­tial treat­ment for a blood dis­or­der known as β-tha­lassemia for those who re­quire reg­u­lar blood trans­fu­sions.

The sec­ond straight unan­i­mous thumbs up opens the po­ten­tial for two FDA ap­provals lat­er this sum­mer for blue­bird — al­though the agency on Fri­day raised some man­u­fac­tur­ing con­cerns for both ther­a­pies.

Ad­comm mem­bers praised the ef­fi­ca­cy of the po­ten­tial gene ther­a­py as “out­stand­ing” and “tremen­dous,” and that the ben­e­fits “def­i­nite­ly out­weigh the risks.” The FDA pre­sen­ter’s fi­nal ben­e­fit-risk as­sess­ment slide made clear that blue­bird’s clin­i­cal stud­ies demon­strate that 89% of sub­jects with TDT who re­ceived beti-cel have achieved trans­fu­sion in­de­pen­dence.

An­drew Oben­shain, CEO of blue­bird, said in a state­ment:

“To­day’s ad­vi­so­ry com­mit­tee rec­om­men­da­tion is recog­ni­tion of the sub­stan­tial body of clin­i­cal da­ta that sup­port beti-cel as a po­ten­tial­ly cu­ra­tive treat­ment op­tion for these pa­tients. We are grate­ful to the mem­bers of the be­ta-tha­lassemia com­mu­ni­ty who con­tributed to to­day’s dis­cus­sion and re­main com­mit­ted to work­ing with the FDA as it com­pletes its re­view of the beti-cel Bi­o­log­ics Li­cense Ap­pli­ca­tion.”

The com­mit­tee on Thurs­day al­so gave a big thumbs up to blue­bird bio’s po­ten­tial gene ther­a­py for the rare but fa­tal con­di­tion known as cere­bral adrenoleukody­s­tro­phy (CALD) by a vote of 15-0, de­spite FDA safe­ty con­cerns.

If both ther­a­pies win ap­proval, the biotech al­so would win two pri­or­i­ty re­view vouch­ers (worth up to about $200 mil­lion to­tal), which could pro­vide des­per­ate re­lief for blue­bird, which laid off about 30% of its work­force in April.

The agency al­so raised con­cerns about the lentivi­ral vec­tor used with beti-cel be­cause of the can­cer cas­es seen de­vel­op­ing in those re­ceiv­ing treat­ment from two oth­er po­ten­tial blue­bird gene ther­a­pies that use a sim­i­lar lentivi­ral vec­tor with a dif­fer­ent safe­ty pro­file. But most of the ad­comm mem­bers found the safe­ty pro­file of beti-cel to be ad­e­quate.

The FDA does not have to fol­low the ad­vice of its ad­comm but it of­ten does.

Ques­tions about a po­ten­tial $2.1 mil­lion price tag for beti-cel al­so might raise some eye­brows, but non­prof­it ICER has called the po­ten­tial gene ther­a­py cost-ef­fec­tive.

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.”

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.