Bristol Myers Squibb's CAR-T liso-cel wins long-delayed FDA nod

After regulatory delays and manufacturing issues caused Bristol Myers Squibb investors to miss out on Celgene contingent value rights, the closely watched CAR-T drug liso-cel has scored an FDA nod. 

Friday, the agency endorsed the drug, to be called Breyanzi, to treat patients with certain types of large B-cell lymphoma who haven’t responded to two other systemic treatments or who have relapsed after receiving those treatments. 

Like other CAR-T drugs, doses of Breyanzi are individually tailored. They’re created using a patient’s own T cells, which are extracted, genetically modified and then infused back into patients to help the body kill lymphoma cells. 

RELATED: Bristol Myers hematology chief Ahmed's out the door amid CAR-T missteps

In a trial of more than 250 patients, 54% of patients who received the CAR-T therapy achieved complete remission. The drug’s label carries a boxed warning for cytokine release syndrome, which can be severe. Because of safety risks, the FDA is requiring centers that administer the drug have a certification indicating staff have been trained and are able to recognize side effects. 

The approval "represents another milestone" for cell and gene therapies, Peter Marks, the director of the FDA's Center for Biologics Evaluation and Research, said in a statement. The therapies "have evolved from promising concepts to practical cancer treatment regimens," he added.

On a conference call earlier this week, BMS' chief commercialization officer Chris Boerner said the company expects the opportunity to launch liso-cel "imminently." The company is "going to be very much focused on ensuring at launch that sites are activated very quickly, that we're able to get patients efficiently moved on to therapy," he added.

Looking forward, the company will look to drive referrals to the medicine and expand the number of sites able to administer it. In the long run, BMS wants to "leverage what we believe to be a differentiated product profile in order to drive brand share," Boerner said.

RELATED: Bristol Myers CVR down the drain as CAR-T drug's FDA manufacturing inspection spots problem 

But while BMS aims to be quick to roll out Breyanzi, the process of getting it approved was anything but. Multiple delays pushed the FDA's decision past the original deadline of mid-August 2020—and ultimately cost investors about $6.4 billion in contingent value rights that came out of BMS' $74 billion Celgene buy.

Nearly 715 million CVRs worth $9 per share were outstanding at the end of the year, and, since BMS didn’t meet all of the CVR requirements, they were worthless when the calendar year flipped to 2021. Aside from an approval for liso-cel, the CVRs also required an FDA approval for multiple myeloma CAR-T med ide-cel by March 31, 2021, and an FDA nod for Zeposia, a multiple sclerosis drug. Zeposia scored its FDA approval last March, and ide-cel is set for an FDA decision by March 27.

Breyanzi will carry a list price of $410,300, a BMS spokesperson said. In pricing the drug, the company "considered many factors including its medical and clinical value, patient value and societal value."