Bluebird New Hope?

The once troubled gene therapy company Bluebird bio recently ushered in good news about its product beti-cel (Zynteglo)

ICER, the full name of The Institute for Clinical and Economic Review, is a well-known independent drug cost regulator in the United States

Despite some doubts, ICER remains bullish on the evidence so far

In its report, Pharmaphorum noted that ICER's review of beti-cel is still ongoing and that the situation may change after an ongoing comment period

And ahead of the August decision date, the beti-cel will be submitted to the FDA advisory committee in June

For the review, ICER delved into 5 beti-cel studies

People with TDT develop life-threatening anemia at about 4 to 6 months of age and must receive monthly blood transfusions as well as additional treatments to prevent complications, such as the use of chelation drugs to remove excess iron from the body

But ICER also issued a warning: "The duration is not long enough to eliminate the uncertainty of whether the effect of the drug will continue to work over a longer period of time

Regarding side effects, ICER noted that promising gene therapies have more or less mild side effects, but few patients experience severe reactions

Bluebird positions beti-cel as a potential treatment, but many thalassemia patients and their families prefer traditional blood transfusions and chelation therapy over current treatments

Still, ICER's approval bodes well for not only Bluebird, but other gene therapy players

However, for a small company like Bluebird, beti-cel is "untenable" for "proper value recognition and market access" in Europe

Faced with an impasse over gene therapy pricing, Bluebird in August planned to "phasing out" beti-cel's European operations, focusing on the U.

Once beti-cel is approved in the United States, beti-cel is expected to tap into a market of 1,000 to 1,500 TDT patients

The fate of beti-cel -- combined with the development of Bluebird's other gene therapies lovo-cel and eli-cel -- could decide whether Bluebird soars or collapses

Last month, Bluebird said its cash flow had been disrupted and expressed serious doubts about its ability to continue as a going concern

The company is currently looking for ways to replenish its funding, which includes the sale of priority review vouchers from beti-cel and eli-cel
.
eli-cel will be reviewed for cerebral adrenoleukodystrophy (CALD) on September 16
.
The drug also encountered regulatory traffic jams in January
.

To stretch its cash flow into the first half of 2023, Bluebird announced earlier this month that it was laying off 30% of its workforce
.
The company had 518 full-time employees as of Jan.
31, according to the company's annual report
.

Original in English: https:// href="https://" target="_blank">https:// references

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