When Bristol Myers Squibb bought Celgene for $ 74 billion in late 2019, one of the centerpieces of the deal was lisa-cel, which hoped to join a small group of CAR-T for non-Hodgkin’s lymphoma. Waiting for approval in 2020, Bristol had its fair share of setbacks by getting flat-cel at the finish line – but now it finally gets the green light.
The FDA on Friday approved Bristol’s Breyanzi (lisocabtagene maraleucel), a CAR-T to treat diffuse large B-cell lymphoma in patients who had previously received two previous rounds of systemic therapy, the agency said in a statement.
The agency based its review on essential Phase I data, showing that 54% of patients treated with lisa-cel achieved complete remission. DLBCL represents about 33% of all non-Hodgkin’s lymphoma cases, said the FDA, of which there are about 77,000 new diagnoses each year.
The single therapy will have a wholesale price of $ 410,300, Bristol said, saying it reached that figure after considering “many factors, including its medical and clinical value, patient value and social value”.
This is a significant prize on Gilead’s Yescarta, which was launched with a $ 373,000 WAC, but a prize on Novartis’s Kymriah and has a $ 475,000 launch WAC.
With approval will come a black box warning label for cytokine release syndrome, a potentially fatal immune system reaction, as well as neurological toxicities. These warnings are serious enough that the FDA has instituted a Risk Assessment and Mitigation Strategy that will require that health facilities and doctors who administer the therapy be specially certified to identify and treat CRS and neuro-toxicities.
These administration notices and restrictions are not exclusive to lisa-cel, however. Yescarta and Kymriah, for example, display the same warnings as the black box, and the unmet clinical need is likely to lead to acceptance in what is an extremely difficult patient population.
Liso-cel is now the third CAR-T on the market for various forms of non-Hodgkin’s lymphoma, along with Yescarta and Kymriah. Bristol intended the therapy to be approved late last year, but delays at Covid-19 and an ugly inspection report at one of the company’s contract factories pushed approval for the new year.
Liso-cel was one of a group of pipeline candidates linked to a $ 9 CVR for the 2019 Celgene Bristol acquisition. With the approval delay, investors missed that payday when the year expired – not a big one a problem for Bristol itself, but definitely a headache for CVR traders.
In December, an FDA inspection at the Lonza Houston plant found a number of problems, including incorrect labeling, products for the U.S. and EU drug markets stored in the same boxes, “poorly maintained” freezing units and expired batches of ingredients. that were not properly discarded, according to a Form 483 letter published online late last month.
Lonza, one of a group of contractors working on flat-cel manufacturing, said delays in the FDA inspection schedule made it difficult to adjust in time for an approval in 2020. Bristol said earlier that it responded to the agency’s concerns within eight days after receiving your letter.