- Bluebird bio said Thursday it doesn't expect to complete an application for U.S. approval of its gene therapy LentiGlobin until mid-2021, as the biotech failed to reach agreement with the Food and Drug Administration about providing additional data for the agency's review.
- The drugmaker had originally planned to finish filing the beta-thalassemia treatment in the first half of this year, but last month pushed that target back to the second half after the FDA requested more information. Further discussions, coupled with the impact of the coronavirus epidemic, will now take more time, Bluebird said.
- Across the Atlantic in Europe, where LentiGlobin is approved as Zynteglo, Bluebird faces more delays in treating its first commercial patient in Germany, which has the third-highest number of COVID-19 cases in Europe. The company now expects the first treatment to occur in the second half of 2020.
Bluebird hinted at larger impacts to come as the coronavirus pandemic added further complexity to issues that had already beset the company prior to the outbreak.
"The company is currently re-evaluating its operating plan" and "will be adjusting priorities and overall expenses," Bluebird said in the Mar. 26 release. The company plans to give investors an update by the time of its first-quarter earnings release.
Discussions between Bluebird and the FDA on LentiGlobin are hung up on data from a type of test called release assays, which are now being validated to support potential commercial use of the therapy. Development of the assays is "likely" to be affected by the coronavirus outbreak, Bluebird said.
With the regulatory situation for LentiGlobin going from "bad to worse," analysts at Piper Sandler wrote in a note to clients they are concerned about the company's cash holdings falling close to one year's worth.
Still, the analysts said they are encouraged that ide-cel, a multiple myeloma therapy under development with Bristol Myers Squibb, is still on track to be submitted to the FDA in the first half of this year.
And Bluebird did provide investors with one potential bit of good LentiGlobin news, saying that a later application for the therapy in beta-thalassemia may allow the company to seek approval for a broader patient population.
But the company anticipates delays in other areas, too. Bluebird expects to seek European Union approval of Lenti-D, a treatment for cerebral adrenoleukodystrophy, by the end of this year, with an application to the FDA in mid-2021. It had previously projected second-half 2020 applications for both the EU and the U.S.
Overall, the pandemic will shift timelines for clinical studies by at least three months, though that will vary by product, Bluebird said. It said planned data disclosures for this year are still on schedule.
COVID-19 is disrupting ongoing studies of Lenti-D for CALD and bb21217 for multiple myeloma, as well as LentiGlobin for both beta-thalassemia and sickle cell disease, the company said. Partner Bristol Myers, meanwhile, has paused enrollment and dosing in three studies of ide-cel.
Bluebird said it's in talks with the FDA about LentiGlobin for sickle cell disease and still expects to give investors a better sense of its regulatory outlook by the end of this year.