Signs are seen outside the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, the United States, August 29, 2020. Portal/Andrew Kelly/File Photo
Aug 17 (Portal) – The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s (BLUE.O) gene therapy for patients with a rare condition that requires regular blood transfusions, and the drugmaker shared the price given a record price of $2.8 million.
The approval sent the company’s shares up 8% and is used to treat beta-thalassemia, which causes a lack of oxygen in the body and commonly leads to liver and heart problems.
The sickest patients, estimated to number as many as 1,500 in the United States, require blood transfusions every two to five weeks.
The therapy, to be branded as Zynteglo, is likely to face some resistance from insurers due to its high price, analysts say.
Gene therapies tend to come with a high price tag as they are often curative and have encountered hurdles in securing insurance coverage.
For example, in 2019 Novartis (NOVN.S) was forced to offer rebates and work out “outcomes-based” installments for its $2.1 million therapy Zolgensma after insurers rejected the drug’s price.
Bluebird has proposed Zynteglo as a potential single-use treatment that could eliminate the need for transfusions, which could result in long-term savings for patients.
The average lifetime cost of transfusions can be $6.4 million, Chief Operating Officer Tom Klima told Portal before the approval. “We believe the prices we are considering still offer significant value to patients.”
Bluebird has been in discussions with insurers about a one-time payment option.
“Possibly up to 80% of that payment will be reimbursed if a patient doesn’t 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 that no such cases had emerged in studies.
Bluebird anticipates that the treatment process for patients will begin in the fourth quarter. However, no revenue from the therapy is expected in 2022 as the treatment cycle from initial cell collection to final transfusion would take an average of 70 to 90 days.
Reporting by Mrinalika Roy in Bengaluru; Editing by Aditya Soni
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