Bluebird to miss Q1 deadline for lovo-cel filing, shares sliced

Bluebird bio had expected to submit a BLA for its sickle cell disease gene therapy lovotibeglogene autotemcel, or lovo-cel, by Friday. Now, that deadline is getting punted for at least a couple of weeks.

Andrew Obenshain

CEO Andrew Obenshain detailed the update during the company’s full-year earnings call Wednesday morning after the gene therapy specialist reported the development in its Q4 and 2022 report.

“We will likely miss the Q1 2023 submission goal,” Obenshain said, noting the application is fully written and ready to go. But bluebird is still waiting on FDA feedback for the company’s CMC module submitted in early March.

Investors took issue with the setback, sending shares of $BLUE down about 30% in morning trading.

This is only the most recent of bluebird’s issues with lovo-cel, formerly known as bb1111. In late 2020, bluebird’s planned filing got a one-year delay after FDA requested the company hand over more data on planned manufacturing processes for the gene therapy.

Obenshain also said Wednesday that after doing product comparability analyses and providing the FDA with a data snapshot in December, the agency came back in February with feedback and some questions, and requested the company respond before filing.

“We anticipate feedback from the FDA within a matter of weeks,” the CEO said. He later declined to specify the questions regulators asked.

Bluebird also said it plans to request priority review in patients 12 and older that have a history of vaso-occlusive events, a common symptom of sickle cell disease that manifests in severe pain and swelling of the hands and feet. If the gene therapy gets approved, a potential commercial launch would be slated for sometime early next year.

Bluebird had said in late 2022, in the wake of a clinical hold being lifted on a lovo-cel trial, that it “remains on track to submit a BLA for lovotibeglogene autotemcel (lovo-cel) in the first quarter of 2023.”

Execs reiterated on the call Wednesday they believe they’ve submitted all the required information necessary to file the BLA, noting that it’s in the FDA’s hands now.

In response to an analyst asking about how this submission differs from previous instances where the FDA raised questions with bluebird’s CMC processes, Obenshain said that FDA and the gene therapy industry “have matured in terms of mutual understanding of what is required for CMC,” additionally noting previous dialogue with the agency about comparability.

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