Bluebird’s $2.8 million gene therapy becomes most expensive drug after U.S. approval
By Mrinalika Roy
(Reuters) -The U.S. Meals and Drug Administration on Wednesday authorized bluebird bio’s gene remedy for sufferers with a uncommon dysfunction requiring common blood transfusions, and the drugmaker priced it at a report $2.8 million.
The approval despatched the corporate’s shares 8% increased and is for the therapy of beta-thalassemia, which causes an oxygen scarcity within the physique and infrequently results in liver and coronary heart points.
The sickest sufferers, estimated to be as much as 1,500 in the US, want blood transfusions each two to 5 weeks.
The remedy, to be branded as Zynteglo, is anticipated to face some resistance from insurers resulting from its steep worth, analysts say.
Gene therapies normally include a excessive price ticket as they’re typically healing and have confronted hurdles in securing insurance coverage protection.
For example, Novartis was in 2019 pressured to supply reductions and work out “outcome-based” installments funds for its $2.1 million remedy Zolgensma after insurers balked on the drug’s worth.
Bluebird has pitched Zynteglo as a possible one-time therapy that might eliminate the necessity for transfusions, leading to financial savings for sufferers over the long run.
The typical price of transfusions over the lifetime will be $6.4 million, Chief Working Officer Tom Klima advised Reuters earlier than the approval. “We really feel the costs we’re contemplating nonetheless deliver a big worth to sufferers.”
Bluebird has been in talks with insurers a couple of one-time cost choice.
“Probably, as much as 80% of that cost might be reimbursed if a affected person doesn’t obtain transfusion independence, they (insurers) are very enthusiastic about that,” Klima stated.
The FDA warned of a possible threat of blood most cancers with the therapy however famous research had no such instances.
Bluebird expects to begin the therapy course of for sufferers within the fourth quarter. No income is, nevertheless, anticipated from the remedy in 2022 because the therapy cycle would take a median of 70 to 90 days from preliminary cell assortment to ultimate transfusion.
(Reporting by Mrinalika Roy in Bengaluru; Modifying by Aditya Soni)
