If all goes according to plan, the first CAR-T cell therapy for multiple myeloma could receive Food and Drug Administration approval by the first quarter of next year.
Cambridge, Massachusetts-based bluebird bio and New York-based Bristol-Myers Squibb said Tuesday that the Food and Drug Administration had accepted and given priority review to their resubmitted application seeking approval for idecabtagene vicleucel, also known as ide-cel or bb2121. The FDA is set to reach a decision on the CAR-T’s approval by March 27, 2021.
The companies filed the application in July, after announcing in May that they had received a refuse-to-file letter from the FDA for the previously submitted application for the therapy due to chemistry, manufacturing and controls issues.
Shares of bluebird (Nasdaq: BLUE) were up more than 1.3% on the Nasdaq in Tuesday afternoon trading following the news, while shares of BMS (NYSE: BMY) were up around 1.4% on the New York Stock Exchange.
As with the original application, the current one is based on results of the Phase II KarMMa study, which tested ide-cel in 128 adults who had previously been treated for multiple myeloma with an immunomodulatory drug, a proteasome inhibitor and a monoclonal antibody targeting CD38 – collectively considered the “backbone” therapies of the disease. Results were presented at the 2020 American Society of Clinical Oncology meeting.
“We are pleased by the significant progress that is being made in partnership with patients and the multiple myeloma community to bring ide-cel to adults with relapsed and refractory multiple myeloma who are triple-class exposed and may benefit from an important new therapeutic option,” BMS senior vice president for cellular therapy development Stanley Frankel said in a statement.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
BCMA has emerged as a popular antigen target for multiple myeloma in recent years. Other CAR-Ts in development that use it include BMS’ in-house orvacabtagene autoleucel and Johnson & Johnson’s JNJ-4528, which uses technology in-licensed from Nanjing, China-based Legend Biotech. Bi-specific antibodies targeting BCMA are in development as well, from J&J and Amgen, while GlaxoSmithKline has the distinction of winning the first approval for an anti-BCMA drug, the antibody-drug conjugate Blenrep (belantamab mafodotin-blmf).
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