FDA Grants Priority Review for Ide-Cel CAR T Therapy
In a recent press release, Bristol Myers Squibb and bluebird bio announced that the FDA has accepted the new drug application for ide-cel (idecabtagene vicleucel, bb2121) CAR T therapy for multiple myeloma and given it priority review.
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021. This is what is required to collect payments for commercial use of a drug or treatment. If ide-cel is approved, it will be the first CAR T therapy available for myeloma patients who have received at least three prior treatments including an immunomodulatory drug (like Revlimid or Pomalyst), a proteasome inhibitor (like Velcade or Kyprolis) and an anti-CD38 antibody (like Sarclisa or Darzalex).
The approval is based on results from the Phase II KarMMa study which used the CAR T therapy in 128 relapsed and refractory multiple myeloma patients from the United States and Europe who had been exposed to the above triple drug classes.
“Today’s acceptance of the BLA for ide-cel for Priority Review by the FDA marks a key moment in our journey to bring this BCMA-directed CAR T cell therapy to multiple myeloma patients who are in desperate need of new options,” said Joanne Smith-Farrell, Ph.D., chief operating officer oncology, bluebird bio. “Based on the body of evidence we have generated in an advanced, heavily pre-treated patient population, our confidence in the potential of ide-cel as an important treatment option remains high. Together with our partners at Bristol Myers Squibb, we are committed to continue working with the FDA to deliver this promising therapy to patients in an expeditious manner.”
Ide-cel was granted Breakthrough Therapy Designation (BTD) by the FDA, and PRIority MEdicines (PRIME) designation and validation of its Marketing Authorization Application (MAA) by the European Medicines Agency for relapsed and refractory multiple myeloma. Bristol Myers Squibb plans regulatory submissions for ide-cel in additional markets outside the U.S. and EU.
Ide-cel is a CAR T therapy that is customized for each patient. T cells are removed through a from the patient through an IV, engineered to target BCMA and expanded, then given back to the patient. BCMA is a new target for a variety of new types of myeloma treatment and is found on most multiple myeloma cells. When Ide-cel recognizes and binds to BCMA, it leads to CAR T cell proliferation, cytokine secretion and killing of BCMA-expressing cells.