With key test still loom­ing, Cel­gene and Ac­celeron nab FDA okay for be­ta-tha­lassemia med

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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

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.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.