Bluebird Bio Offers World's Most Expensive Drugs, But Uncertainty Looms Over Its Financial Future

Bluebird Bio Inc BLUE is about to become the seller of the two most expensive drugs in the U.S. and probably the world. The price tag of $3 million is already facing backlash.
Two FDA approvals this year might have saved the company from the financial abyss. But Bluebird will be cash-strapped for a while, the WSJ reported.
In April, Bluebird laid off around 30% of its workforce.
In September, the FDA approved Skysona to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
Related: FDA Okays bluebird bio's $3M Gene Therapy For Rare Neurological Disorder In Boys: What Investors Should Know
In August, FDA approved Bluebird's Zynteglo (beti-cel), a one-time gene therapy custom-designed for beta‑thalassemia.
Related: Bluebird's Gene Therapy Scores US Approval For Genetic Blood Disorder, Probably The Most Expensive
Beta thalassemia and CALD afflict tiny portions of the population, limiting their economic potential.
The report cited an analyst at RBC Capital Markets that with only an estimated 1,300 people with transfusion-dependent beta-thalassemia in the U.S., sales for the two treatments might reach just over $200 million by 2030.
The company expects 2022 cash burn of almost $340 million. As of the second quarter, it had about $218 million in cash and equivalents.
The company "has a clear path to financial sustainability and considerable positive momentum as we approach the anticipated BLA filing for lovo-cel for sickle cell disease in Q1 2023," the report cited a spokeswoman.
While approval certainly would boost the company's prospects, it could still face competition from other gene therapy players like Vertex Pharmaceuticals Inc VRTX and CRISPR Therapeutics AG CRSP.
Price Action: BLUE shares are down 3.47% at $6.11 on the last check Monday.

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