- The Food and Drug Administration began a priority review of Bluebird bio’s gene therapy for sickle cell disease and will issue a decision by Dec. 20, the company said Wednesday.
- The deadline for an answer puts Bluebird just days behind Vertex Pharmaceuticals and CRISPR Therapeutics, which expect to hear on their own sickle cell application by Dec. 8. Vertex and CRISPR are angling to sell the first medicine based on CRISPR gene editing technology.
- Bluebird’s one-time therapy, lovo-cel, aims to treat the underlying cause of the disease by giving patients functional copies of a gene that can reduce the number of “sickled” blood cells. Those misshapen and hardened red blood cells can cause painful episodes of blocked blood flow, or vaso-occlusion.
While Bluebird already has two approved gene therapies, investors are focused on lovo-cel as its most promising product. Bluebird is seeking approval of the therapy for patients who are at least 12 years old and have a history of vaso-occlusive events; it estimates that about 20,000 people in the U.S. would be eligible for the treatment.
Even if the company can only win over a relatively small percentage of that group, lovo-cel has the potential to yield sales of $2 billion or more, based on its expected price tag, Baird analyst Jack Allen wrote in a note to clients Wednesday. The company priced its two approved gene therapies at $2.8 million and $3 million per patient.
To date, Bluebird has brought in revenue largely from the sale of priority review vouchers it received after winning approval of its other two products, Zynteglo for beta-thalassemia and Skysona for cerebral adrenoleukodystrophy. It sold one voucher to the Dutch biotech Argenx for $102 million and another to Bristol Myers Squibb for $95 million.
Meanwhile, it’s slowly building the infrastructure to bring in sales from its gene therapies by activating qualified treatment centers. The company this year announced the first patient infusions with Zynteglo and Skysona, and a number of other patients have begun the cell collection process before treatment.
The work already invested in Zynteglo and Skysona may help Bluebird compete with Vertex and CRISPR if the two rival therapies win approval at about the same time, Baird’s Allen said. A quick launch would be welcome news to Bluebird investors, who have seen the company’s stock drop to below $4 after flying above $150 in March 2018.
A big factor in the stock drop has been a series of setbacks for lovo-cel. The company temporarily stopped two studies of the therapy in early 2021 amid concerns about a trial participant who developed leukemia and another who had a cancer-like disease of the bone marrow. Then, the FDA hit Bluebird with another clinical hold in late 2021 after a younger study volunteer developed persistent anemia.
After getting its studies back on track, Bluebird originally aimed to submit its application for lovo-cel by the end of March but got sidetracked waiting for FDA feedback on its manufacturing processes. The company finally filed in April.