The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
Gene therapy can offer hope to patients with previously untreatable diseases, but there’s more work to be done to improve both the safety and efficacy of this therapeutic modality.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
Bluebird Bio gene therapy Skysona has won FDA approval, making it the first FDA-approved therapy for cerebral adrenoleukodystrophy, an ultra-rare and fatal neurological disorder. Skysona’s regulatory green light follows the August approval of Zynteglo, a Bluebird gene therapy for a rare blood disorder.
FDA approval of Bluebird Bio’s Zynteglo makes it the first gene therapy in the U.S. for the rare blood disorder beta thalassemia. The biotech company set a $2.8 million price for the one-time treatment, but it also came up with a reimbursement strategy designed to ease the financial risk to payers.
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.
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, CEO Nick Leschly said the company has asked the FDA for permission to resume the three clinical trials that were placed on hold in February during the inquiry.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
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.
The companies said Tuesday that the FDA had accepted their application for idecabtagene vicleucel, which they resubmitted in July after the agency initially refused to file it in May. The action date is in March of next year.
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.
Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
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Data on multiple CAR-Ts presented at the recent ASCO meeting have raised the prospect of outpatient use of the therapies, which is favorable for reimbursement as well as for patients. But experts pointed to significant infrastructure challenges that centers face.
The FDA requested supplemental documentation related to chemistry, manufacturing and controls rather than new clinical or preclinical data. The companies plan to resubmit the application and request a priority review by the end of July.
BMS bought out its milestone and royalty obligations for ex-U.S. markets for idecabtagene vicleucel, or ide-cel, which is currently undergoing FDA review. The companies will continue to share profits and losses in the U.S.
The submission of an application for idecabtagene vicleucel comes shortly after Bristol-Myers Squibb filed for approval of another CAR-T, lisocabtagene maraleucel, in an aggressive form of lymphoma. The FDA is expected to rule on that CAR-T by February.
Intercept Pharmaceuticals and bluebird bio said they had encountered regulatory snags at the FDA for their investigational drugs, while Pfizer is pausing most of its clinical trials and delaying completion of a deal with Mylan to create a new company.