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.
“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.