Category: bluebird bio
bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. By the end of the week, it was two for two, with both recommended for approval by a unanimous vote. Thursday saw the Cellular, […]
The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. Members of the agency’s Cellular, Tissue and Gene Therapies advisory committee voted 15 to 0 in favour of approval of elivaldogene autotemcel – also known as eli-cel […]
US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. ICER’s assessment is still at the draft stage and could be changed after an ongoing comment period, but will be […]
Gene therapy specialist bluebird bio has said 30% of its workforce will lose their jobs, in a bid to conserve cash as it tried to get its lead programmes over the finish line. The biotech said it wants to eke out its financial reserves while it waits for the FDA’s verdict on betibeglogene autotemcel (beti-cel) […]
As bluebird bio struggles to get its gene therapies over the finishing line, the company says its financial position raises “substantial doubt” about its ability to continue as a going concern. The biotech is sitting on around $442 million in liquid assets, but is expecting its cash burn in 2022 to be around $400 million, […]
Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. On the firm’s second-quarter results call, Obenshain said the biotech will start “an orderly wind-down” […]
Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The EMA has cleared Skysona (elivaldogene autotemcel) for use in CALD patients aged under 18 who have an ABCD1 genetic mutation and no matched […]
In an interview with PharmaShots, Rich Colvin, M.D., Ph.D., Interim Chief Medical Officer at bluebird bio shares his views on the Skysona and its working on CALD patients. Shots: If granted marketing authorization, Skysona would be the first approved one-time gene therapy for CALD which is a rare neurogenerative disease with typically childhood onset The […]
The EU medicines regulator has said that there is some evidence to suggest a possible link between mRNA-based vaccines for COVID-19 and rare cases of heart inflammation. According to the EMA’s pharmacovigilance risk assessment committee (PRAC), labelling for Pfizer/BioNTech’s Comirnaty and Moderna’s Spikevax should be updated to reflect “very rare cases” of myocarditis and pericarditis […]
Johnson & Johnson has secured a six-month FDA review for its multiple myeloma CAR-T therapy ciltacabtagene autoleucel (cilta-cel), narrowing the lead for Bristol-Myers Squibb and bluebird bio’s rival Abecma. The US regulator is now scheduled to complete its review of cilta-cel by 29 November for adults with relapsed and/or refractory multiple myeloma who have previously […]
The UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – but there are still many barriers to wider adoption. These challenges often arise because these therapies go through the same value assessment processes as less complex medicines, and the infrastructure […]
US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has given the go-ahead to bluebird’s therapy – known as Skysona (elivaldogene autotemcel) […]
The FDA has approved a Bristol Myers Squibb cell therapy made by engineering a patient’s immune cells to target a cancer protein called BCMA. The regulatory decision for Abecma in multiple myeloma gives BMS its second approved CAR-T therapy.
Shots: The approval is based on the P-II KarMMa trial that involves assessing Abecma in 127 patients with r/r MM prior treated with 3L+ therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 Ab Results: ORR (72%); sCR (28%); mDoR (11mos.); responses were rapid and durable, with a median time to response of […]
bluebird bio has announced new clinical trial data from its gene therapy for patients with cerebral adrenoleukodystrophy (CALD), including long-term follow-up data. CALD is the most severe form of adrenoleukodystrophy (ALD), a rare, X-linked metabolic disease affecting 1 in 21,000 newborn boys worldwide. Approximately 40% of boys with ALD will develop CALD1, typically between the […]
Bluebird bio said independent analyses found that the gene therapy inserted its payload at a site not known to cause cancer. Based on the results, the company has asked the FDA for permission to resume all of the clinical trials that were placed on hold in February during the inquiry.
bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, […]
Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. That’s the view of analysts at Jefferies, who suggest it could “re-ignite concerns over the […]
Cancer is a known risk of lentiviral-based gene therapies. Though bluebird says its gene therapy is engineered to avoid that risk, the company is suspending two clinical trials pending the outcome of its inquiry.
It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. NICE is assessing Zynteglo (betibeglogene autotemcel), a one-off gene therapy for the condition, which can have life-threatening consequences and is associated with a curtailed […]
Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor […]
Shots: Bluebird plans to separate its severe genetic disease & oncology businesses into differentiated and independent publicly traded companies. The company will retain focus on SGD and will launch its oncology business (“Oncology Newco”) as a new entity The spinoff is expected to result in two independent, publicly traded companies by the end of 2021 […]
US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins […]
bluebird bio has presented long-term data from its Zinteglo one-time gene therapy for the blood disorder beta-thalassaemia, as the company continues talks with payers in Europe to bring the ultra-pricey treatment to market. The European Medicines Agency (EMA) has granted a conditional marketing authorisation for the drug that will be marketed as Zinteglo (betibeglogene autotemcel), […]
Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD […]
The companies’ resubmission of their application Wednesday seeking approval for idecabtagene vicleucel was in line with the timeline they provided in May, when the FDA sent a refuse-to-file letter in response to their initial submission.
Bristol-Myers Squibb and bluebird bio have refiled their CAR-T therapy for multiple myeloma, leaving the outcome of a three drug “bet” with former Celgene shareholders dependent on a fast review from the FDA. BMS gave Celgene’s shareholders a “Contingent Value Right” (CVR) to sweeten the $74 billion merger last November. Each shareholder got one CVR […]