The FDA has placed a nugget of positive clinical news into bluebird bio’s stocking, lifting a year-old partial clinical hold of its sickle cell gene therapy for patients under 18.
The regulatory wave-through, announced Monday, follows an investigation into a case of anemia among one of the patients treated with lovo-cel, spurring a hold on the trial in December 2021. The company’s assessment, which also included a similar case in the adult trial, found that the two cases were linked to a specific gene mutation. As a result, the company added the genotype to its inclusion criteria.
“We are working closely with study investigators and clinical trial sites to resume enrollment and treatment of pediatric and adolescent patients in the first quarter of next year,” said bluebird’s chief medical officer Richard Colvin, M.D., Ph.D. The adult trial has continued to run without issue, with the company planning to file for approval in early 2023.
The news caps off a generally successful year for the company, marked by the FDA’s approval of its $2.8 million beta-thalassemia treatment, Zynteglo. The FDA also granted accelerated approval to Skysona, bluebird’s treatment for cerebral adrenoleukodystrophy, a genetic brain injury that damages myelin sheaths.
And with its sickle cell gene therapy fast approaching its own approval application, bluebird can now turn its clinical attention toward the adolescent population. The question now is, what’s next?
Beyond lovo-cell, bluebird has just one additional clinical-stage med, a runner-up sickle cell treatment targeting the BCL11A gene. The company says it has multiple preclinical targets that it’s cooking up, though details remain sparse.
But the company was not spared from a tumultuous market in 2022 that battered much of the industry, cutting 30% of its staff in April to save costs. Even after the layoffs, the biotech’s R&D spend was north of $50 million in the third quarter, a significant price tag given the company’s $141 million in cash and equivalents. Bluebird was able to cushion that position last month, however, selling a priority review voucher for $102 million.