Bristol Myers Squibb’s bid to win FDA approval for its multiple myeloma cell therapy has hit a detour. The FDA declined to review the experimental treatment and has instead asked the company to provide more information.
Bristol (NYSE: BMY) announced Wednesday that it received a “refusal to file” letter for the experimental therapy, idecabtagene vicleucel (ide-cel). Such letters inform companies of deficiencies in an application and point to what a company must do to address them. In this case, Bristol and partner bluebird bio (NASDAQ: BLUE) say the regulator has questions about how the product is made.
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The FDA is not asking the companies to conduct additional clinical testing of ide-cel, Bristol CEO Giovanni Caforio said on a Wednesday conference call. The data are information that the companies already have and the goal is to resubmit the application by the end of July, he added.
Muliple myeloma is a cancer of the plasma cells, which are found in bone marrow. Ide-cel is one of a number of experimental therapies that aim to treat multiple myeloma by targeting B cell maturation antigen (BCMA), a protein found in abundance on the surface of multiple myeloma cells, plasma cells, and some mature B cells. The therapy is made by drawing T cells from a patient, engineering them to recognize BCMA, and then infusing them back into the patient to do their work. The engineering portion of the process uses lentivirus to alter the T cells.
In a Wednesday regulatory filing, Cambridge, MA-based bluebird states that the FDA asked for more information “detailing the validation and control processes used in the lentiviral vector and drug product manufacturing processes for ide-cel.”
When Bristol submitted the ide-cel application to the FDA in late March, the company believed that it was complete, Caforio said on the call. He declined to go into specific detail about the FDA’s questions but he said the information requested is typically part of the review process rather than the initial submission. Speaking on a separate conference call, bluebird CEO Nick Leschly said that the regulator is asking for additional documentation and the companies will not need to do any additional tests of the manufacturing process.
Ide-cel was added to the Bristol pipeline last year via the $74 billion acquisition of Celgene. The Summit, NJ, company was developing the cell therapy in partnership with bluebird. The Celgene acquisition was structured to pay its shareholders more if three of the company’s drugs win FDA approval. But this “contingent value right” will be paid only if all three pass regulatory muster. The first, multiple sclerosis drug ozanimod, was approved last month. The second, lisocabtagene maraleucel, a cell therapy developed for large B-cell lymphoma, is expected to receive an FDA decision in August. If that drug is approved, the additional payment to Celgene shareholders kicks in only if ide-cel secures a favorable regulatory decision by March 31, 2021, according to the deal terms.
A number of other companies are also in the running to develop and commercialize BCMA-targeting cell therapies for multiple myeloma. Novartis (NYSE: NVS) is in the hunt through a partnership with Poseida Therapeutics. Nanjing Legend Biotech is developing a cell therapy under an alliance with Johnson & Johnson (NYSE: JNJ).
Meanwhile, Amgen (NASDAQ: AMGN) is developing an antibody drug that binds to both T cells and BCMA. GlaxoSmithKline (NYSE: GSK) submitted its antibody drug conjugate, belantamab mafodotin, for FDA review in January, giving it the edge in the race to commercialize the first anti-BCMA multiple myeloma treatment.
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