"Bristol Myers Squibb, bluebird land once-high-stakes approval...

"Bristol Myers Squibb, bluebird land once-high-stakes approval on BCMA CAR-T — and it bodes well for rival J&JAmber TongSenior Editor

Almost a year after getting slammed by a surprise refuse-to-file letter, Bristol Myers Squibb and bluebird have been cleared to market the first BCMA CAR-T therapy.Once a closely-watched event, the approval of ide-cel in multiple myeloma received a relatively muted welcome when it was announced Friday afternoon.
It was largely expected by analysts, who were however surprised by parts of the label.The drug, now dubbed Abecma, will sport a list price of $419,500.
The OK marked a much-needed victory — coming on the heels of another win on liso-cel, or Breyanzi — for both companies after regulatory setbacks that led to the overnight evaporation of a $6 billion CVR that Celgene investors got from Bristol and serious credibility issues for bluebird.
“Bristol Myers Squibb is now the only company with two approved CAR T cell therapies with distinct targets of CD19 and BCMA,” said Bristol CMO Samit Hirawat.
The pharma giant will now split promotion costs and profit with the yet-unnamed oncology newco that bluebird is spinning out as CEO Nick Leschly separates the rare disease and cancer pipelines.Regulators, who had spurned the initial BLA because it included a faulty section on CMC, gave thumbs up to the data from the pivotal Phase II KarMMa trial. Among 100 patients taking the full dose of ide-cel, 72% saw their tumor shrink — and 28% achieved a stringent complete response. An estimated 65% of those had remission lasting at least 12 months, bolstering Bristol’s claim to a durable effect. Median time to response was just 30 days, they added.“We were surprised by a few aspects of the label: Abecma was approved for 5L+, versus our expectation of 4L+, and HLH was included as a black box warning,” Stifel analyst Benjamin Burnett wrote. “However, after reflecting on these points with a KOL (tertiary care physician who leads CAR-T efforts at his institution), we think neither will significantly impact adoption.”

Biren Amin of Jefferies, meanwhile, noted that the approved dose range of 300-400 million cells was narrower than expected. He added that the approval bodes well for J&J-partnered Legend Biotech, whose BCMA CAR-T, cilta-cel, has a PDUFA date lined up for this year.Still, Bristol has been throwing its weight behind ide-cel, so confident about its status as a frontrunner that it ejected a second BCMA CAR-T candidate (orva-cel, originally developed by Juno) from the pipeline, writing off $470 million in the process.At least some investors were energized, bidding bluebird shares $BLUE up 8.27% to $32.46."

The above is the write for the latest Car T approved by the FDA. Note only a 28% CR rate, 72% with partial response. 65% of patients having remission of at least 12months. Approval after a phase 2 trial of 100 patients. Treatment target cost $US 419500.