Lonza says observations made by the FDA at its Texas facility should not further hamper the already delayed approval of Bristol-Myers Squibb’s lymphoma CAR-T candidate liso-cel.
Bristol-Myers Squibb (BMS) added lisocabtagene maraleucel (liso-cel), an autologous anti-CD19 chimeric antigen receptor (CAR) T‑cell targeting lymphoma, to its pipeline through its $74 billion acquisition of Celgene.
Though a Biologics License Application (BLA) was submitted to the US Food and Drug Administration (FDA) in December 2019, COVID-19-related constraints pushed back regulatory inspections necessary for approval meaning an already extended PDUFA action date of November 16 2020 was missed.
BMS had hoped the approval would come by the end of the year in order to fulfil one of the required remaining milestones of the Contingent Value Rights issued upon the close of the Celgene acquisition late last year, but in a statement issued last week the firm told stakeholders “the security will no longer trade on the NYSE, and the CVRs are no longer eligible for payment.”
The company had seen a setback following the FDA’s visit to its Bothell, Washington facility, finally inspected in October, when it received a Form 483 citing six observations. But observations at third-party viral vector manufacturer Lonza’s Texan plant noted during an inspection in December are also causing a headache in achieving approval.
“We will continue to work closely with the FDA to bring this therapy to patients as quickly as possible,” BMS said in a statement last week.
The Houston, Texas facility was described as the world’s largest dedicated cell-and-gene-therapy facility when it opened in April 2018.
According to a Lonza spokeswoman, the firm was “ready to host the PAI (Pre Approval Inspection) in line with the request from the FDA for an April 2020 visit,” but COVID-19-related precautionary measures pushed this back eight months.
“The FDA made a limited number of observations at the close of the inspection to which Lonza and BMS expeditiously responded within eight days (well within the required fifteen days),” we were told. “We cannot comment on the issues raised but we are not aware of any reasons why the observations would delay approval.”
She added “the limited number of observations were specific to the manufacturing of the viral vector for liso-cel,” and no other programs are affected.
“We highly value all feedback from regulatory agencies, applying lessons learned to continuously improve our operational expertise. We are committed to supporting BMS in their goal of bringing liso-cel to patients with relapsed or refractory large B-cell lymphoma.”