Bluebird's $2.8 million gene therapy becomes most expensive drug after
U.S. approval
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[August 18, 2022]
By Mrinalika Roy
(Reuters) -The U.S. Food and Drug
Administration on Wednesday approved bluebird bio's gene therapy for
patients with a rare disorder requiring regular blood transfusions, and
the drugmaker priced it at a record $2.8 million.
The approval sent the company's shares 8% higher and is for the
treatment of beta-thalassemia, which causes an oxygen shortage in the
body and often leads to liver and heart issues.
The sickest patients, estimated to be up to 1,500 in the United States,
need blood transfusions every two to five weeks.
The therapy, to be branded as Zynteglo, is expected to face some
resistance from insurers due to its steep price, analysts say.
Gene therapies usually come with a high price tag as they are often
curative and have faced hurdles in securing insurance coverage.
![](http://archives.lincolndailynews.com/2022/Aug/18/images/ads/current/lincolnag_sda_FAIR_2022.png)
For instance, Novartis was in 2019 forced to offer discounts and work
out "outcome-based" installments payments for its $2.1 million therapy
Zolgensma after insurers balked at the drug's price.
Bluebird has pitched Zynteglo as a potential one-time treatment that
could do away with the need for transfusions, resulting in savings for
patients over the long term.
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Signage is seen outside of the Food and
Drug Administration (FDA) headquarters in White Oak, Maryland, U.S.,
August 29, 2020. REUTERS/Andrew Kelly/File Photo
![](http://archives.lincolndailynews.com/2022/Aug/18/images/ads/current/chicagostreet_lda_FAIR_2022.png) The average cost of transfusions
over the lifetime can be $6.4 million, Chief Operating Officer Tom
Klima told Reuters before the approval. "We feel the prices we are
considering still bring a significant value to patients."
Bluebird has been in talks with insurers about a one-time payment
option.
"Potentially, up to 80% of that payment will be reimbursed if a
patient does not achieve transfusion independence, they (insurers)
are very excited about that," Klima said.
The FDA warned of a potential risk of blood cancer with the
treatment but noted studies had no such cases.
Bluebird expects to start the treatment process for patients in the
fourth quarter. No revenue is, however, expected from the therapy in
2022 as the treatment cycle would take an average of 70 to 90 days
from initial cell collection to final transfusion.
(Reporting by Mrinalika Roy in Bengaluru; Editing by Aditya Soni)
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