Bristol Myers Squibb gets long-awaited FDA nod for liso-cel, joining shortlist of CAR-T lymphoma winners – Endpoints News



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When Bristol Myers Squibb bought Celgene for $ 74 billion at the end of 2019, one of the centerpieces of that deal was liso-cel, who hoped to join a small group of CAR-Ts for non-Hodgkin lymphoma. Expecting approval in 2020, Bristol had its share of setbacks to get liso-cel across the finish line – but now it finally has 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 have already received two cycles of systemic therapy, the agency said in a statement.

The agency based its review on pivotal Phase I data showing that 54% of patients treated with liso-cel achieve complete remission. DLBCL accounts for about 33% of all non-Hodgkin lymphoma cases, the FDA said, including about 77,000 new diagnoses each year.

The one-time therapy will be offered at a wholesale price of $ 410,300, Bristol said, claiming to have found that figure after taking into account “many factors including its medical and clinical value, patient value and societal value. . “

That’s a significant bounty over Gilead’s Yescarta, which launched with a WAC of $ 373,000, but a bounty on Novartis’ Kymriah, which launches a WAC of $ 475,000.

With approval will come a black box warning label for cytokine release syndrome, a potentially fatal overreaction of the immune system, as well as for neurological toxicities. These warnings are severe enough that the FDA has instituted a risk assessment and mitigation strategy that will require healthcare facilities and physicians administering therapy to be specially certified to identify and treat CRS and neurotoxicity.

These warnings and administration restrictions are not, however, unique to liso-cel. Yescarta and Kymriah, for example, sport the same black box warnings, and the unmet clinical need will likely lead to adoption in what is an extremely difficult patient population to treat.

Liso-cel is now the third CAR-T in the market for various forms of non-Hodgkin lymphoma, alongside Yescarta and Kymriah. Bristol aimed to get the therapy approved late last year, but delays from Covid-19 and a horrific inspection report at one of the company’s contract manufacturing sites pushed approval into the new Year.

Liso-cel was among a group of pipeline candidates tied to a $ 9 CVR of Celgene’s takeover by Bristol in 2019. With the approval delay, investors lost that payday when the year expired – not a major problem for Bristol itself but certainly a headache for CVR traders.

In December, an FDA inspection at the Lonza Houston plant revealed a range of issues, including labeling errors, products destined for the US and European drug markets stored in the same bins, freezer units “Poorly maintained” and lots of expired ingredients that were not properly disposed of, according to a Form 483 letter posted online late last month.

Lonza, one of a group of contractors working on manufacturing liso-cel, said delays in the FDA inspection schedule made it difficult to adjust in time for approval by 2020. Bristol had previously said it responded to the agency’s concerns within eight days of receiving its letter.

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