Legend's status put on hold as FDA delays Janssen-partnered CAR-T decision

GenScript Biotech - Legend Biotech
The FDA hit the brakes as it has questions about Legend Biotech and Johnson & Johnson's BCMA-directed CAR-T therapy.

Legend Biotech and Johnson & Johnson have been hit by an FDA delay for their cell therapy blood cancer hopeful ciltacabtagene autoleucel.

It wasn’t supposed to be like this: Chinese biopharma Legend and U.S. partner Janssen, the R&D unit of J&J, were the darlings of the American Society for Clinical Oncology annual meeting a few years ago, with their CAR-T, also known as cilta-cel, wowing the crowd, leading to a speedy review by the FDA earlier this year in relapsed/refractory multiple myeloma.

They had a sales team and marketing platforms in place, ready to jump into action as soon as that expected green light came. But for now, it’s at yellow for the next four months.

RELATED: Johnson & Johnson preps for first CAR-T launch with a new patient support program, a dedicated sales team and more

The FDA hit the brakes as it has questions about the BCMA-directed CAR-T therapy that need time to resolve: The original PDUFA date under its priority review had been in late November, but, now, the U.S. agency has pushed this back to Feb. 28 next year.

Why? Well, that’s all a little vague. In a brief statement, the pair said the delay was to give it “sufficient time to review information recently submitted pertaining to an updated analytical method following an FDA information request.”

Legend and Janssen met with the FDA Nov. 1, though they are not sharing details of what happened. They did add, however, that no “additional clinical data” have been requested.

Analysts at Jefferies have a little extra color after speaking to the company, saying in a note to clients that the FDA information request "pertains to the CMC section and the method validation for final products," though it added that this "was not related to the manufacturing process or the pre-approval inspection." The firm is still confident of approval and sees little long-term impact on the companies from the delay.

J&J and Legend based their FDA application on an open-label phase 1b/2 study in adults with relapsed and/or refractory multiple myeloma who had tried three previous therapies. After one year, 97% of patients responded, and 67% showed no signs of cancer.

Those data attracted attention from leading multiple myeloma experts. After speaking with a specialist at a “leading academic center,” RBC Capital Markets analysts wrote to clients back in March that the doctor was impressed by the “deep and durable” responses.

J&J's drug is also associated with higher toxicity, the expert noted, so she envisioned using it in healthier patients to start.

RELATED: ASCO: J&J's anti-BCMA CAR-T pads its case ahead of speedy review and Bristol Myers showdown

Bristol Myers Squibb and bluebird bio scored an FDA nod for their multiple myeloma CAR-T drug Abecma back in Marc,h and Janssen/Legend were hoping to draft them to the finish line, but now have longer to wait. Abecma also had issues en route to approval, being hit with a refuse to file letter, though, in the end, this was swiftly resolved.

“We are working closely with Janssen and the FDA to facilitate an efficient and thorough review of the BLA for cilta-cel,” said Ying Huang, Ph.D., CEO and chief financial officer at Legend.

“We remain confident that cilta-cel has shown great promise in patients with relapsed and refractory multiple myeloma, and we are focused on making this therapy available to them in the U.S. as soon as possible.”

Legend’s shares on the Nasdaq were off nearly 9% in premarket trading Tuesday morning.