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

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  • 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).
  • RelatedFDA 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.
  • RelatedBluebird'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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