While pric­ing con­cerns con­tin­ue to cloud the fu­ture of blue­bird bio’s gene ther­a­py fix, the FDA has giv­en its okay for Cel­gene and Ac­celeron’s be­ta-tha­lassemia drug Re­blozyl — to treat ane­mia in adults who re­quire reg­u­lar trans­fu­sions. An ap­proval for myelodys­plas­tic syn­dromes (MDS) — which will like­ly de­ter­mine if the drug is a ma­jor com­mer­cial suc­cess — is still un­der re­view.

The drug will be priced at $3,441 per 25 mg vial, Ac­celeron said.

A fu­sion pro­tein, Re­blozyl (lus­pa­ter­cept-aamt) boosts red blood cell growth in ane­mic pa­tients by tar­get­ting TGF-be­ta pro­teins that play a role in these cells’ mat­u­ra­tion. The drug, which had been grant­ed break­through sta­tus, was ap­proved on the ba­sis of a 336-per­son Phase III tri­al.  The tri­al showed slight­ly over a fifth of the be­ta-tha­lassemia pa­tients who took the drug saw a 33% or greater re­duc­tion in trans­fu­sion bur­den.

Richard Paz­dur FDA

“When pa­tients re­ceive mul­ti­ple blood trans­fu­sions, there is a risk for iron over­load, which can af­fect many or­gans,” said FDA on­col­o­gy chief Richard Paz­dur. “To­day’s ap­proval pro­vides pa­tients with a ther­a­py that, for the first time, will help de­crease the num­ber of blood trans­fu­sions.”

Cel­gene ac­quired the drug in 2011 for $25 mil­lion up­front and a mod­est $217 mil­lion in mile­stones. It’s a key part of the late-stage pipeline Bris­tol-My­ers Squibb bet on in their $74 bil­lion buy­out; an­a­lysts have pegged the drug as a po­ten­tial block­buster worth up to $2 bil­lion per year.

Two thirds of that pro­ject­ed rev­enue, though, come from MDS pa­tients.

For the be­ta-tha­lassemia pa­tients, a dif­fer­ent so­lu­tion emerged in the time since Cel­gene’s deal as blue­bird bio de­vel­oped a gene ther­a­py that could of­fer a com­plete rem­e­dy for the dis­ease. But that ther­a­py, Zyn­te­glo, has yet to be ap­proved in the US (al­though an FDA okay could come as soon as next year), and in Eu­rope, where the EMA has giv­en its bless­ing, the promise of a one-shot fix for a life­long dis­ease has been com­pro­mised by a $1.8 mil­lion price tag — twice what an­a­lysts had pro­ject­ed for the drug on the con­ti­nent.

The FDA is due to rule on the MDS in­di­ca­tion by April 4, 2020. Cel­gene ap­plied on the ba­sis of late-stage da­ta that showed 38% of pa­tients went 8 weeks with­out need­ing a blood trans­fu­sion and 28% man­aged 12 weeks with­out one.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.