In the past two days, the FDA advisory committee is voting on the two gene therapies of Bluebird, Beti cel and Eli cel. Although the documents released on Tuesday showed that scientists cautiously supported Beti cel (zynteglo) therapy and were skeptical of Eli cel, a gene therapy for brain wasting diseases. However, for "does Eli cel benefit more than risk in any subgroup of children with early cerebral adrenoleukodystrophy?" The voting results of ctgtac members were 15 in favor and 0 against.
Despite the affirmative vote, it is still unknown how FDA will make a final decision. FDA will make a final decision on the approval of Beti cel and Eli cel on August 19 and September 16, respectively.
Its decision will determine the fate of Bluebird, shape the research and development methods of other gene therapies, and more likely change the treatment methods of two genetic diseases.
Now, after years of research and billions of dollars of investment, Bluebird biology has a shortage of funds, or led to the bankruptcy of the company within a year. If these two gene therapies are approved, it means that Bluebird organisms will continue to survive, which is also conducive to the development of gene therapy for rare diseases.
Before that, Beti cel and Eli cel obtained milestone approvals in Europe, but manufacturing obstacles and difficulties in obtaining reimbursement forced Bluebird to withdraw from the market and close its business there. Only a few patients received both treatments. In the United States, disagreements with the FDA on production testing twice delayed Bluebird's plan to seek beta cel approval, and slowed the progress of another gene therapy for sickle cells in blood conditions. FDA initially rejected Bluebird and partner Bristol Myers Squibb's application for the third drug mentioned by leschly, a cell therapy for multiple myeloma, which was finally approved in early 2021.
Delays and setbacks are especially costly for Bluebird. Its market value was close to US $10billion at the beginning of 2018, and has now dropped to just over US $250million. The company will move its headquarters from Cambridge to Somerville, Massachusetts, to save money, and said in April that it would lay off about one-third of its employees.
However, cost cutting is only a drop in the bucket. Bluebird lost hundreds of millions of dollars in the quarter of this year, and the available funds are only 267million dollars. Everyone speculates whether it can make a living next year.
Therefore, the survival of Bluebird depends on whether FDA Approves Beti cel and Eli cel. The approval of both will enable Bluebird to make money not only from commercial sales, but also from the priority review certificate granted by FDA, which is usually worth about $100million.
Security concerns
However, Bluebird's gene therapy may not be approved by FDA because they have had safety problems in the past year.
In february2021, a clinical trial on sickle cells was announced to be suspended because two treated patients developed leukemia like cancer. Subsequently, the FDA stopped the clinical trial.
Later, the company found that there was no clear link between leukemia cases and their treatment. In June 2021, FDA cancelled its mandatory suspension of trials.
But then in August, the FDA suspended the clinical trial of Eli cel because Bluebird reported a bone marrow cancer with myelodysplastic syndrome in a patient receiving Eli cel gene therapy. Subsequently, two patients receiving Eli cel gene therapy were diagnosed with acute myeloid leukemia and myelodysplastic syndrome (MDS). Two of them were directly related to treatment, and the other one was considered to be highly likely related to treatment.
Benefits and risks
In view of this situation, FDA and its consultants have to weigh the safety risks and benefits of Bluebird gene therapy.
FDA seems to be more supportive of Beti cel, which makes β Patients with thalassemia do not need regular blood transfusion, which reduces the incidence of complications related to chronic blood transfusion. Even so, the FDA asked its consultants to weigh the cancer risk of patients observed in Bluebird's Eli cel therapy and sickle cell therapy trials.
summary
If Bluebird's gene therapy is approved, Eli cel and Beti cel will become the third and fourth genetic disease gene therapy approved in the United States after Roche's luxturna and Novartis' zolgensma.
Despite the booming investment in gene therapy, many companies are restructuring their research, cutting costs or laying off staff in the face of the market downturn. So the market is in urgent need of a good news to stimulate development. Let's look forward to the approval result of FDA
