FDA Allows Bluebird Bio To Resume Sickle Cell Disease Studies In Patients Below 18, With Changed Protocol

  • The FDA removed its partial clinical hold on Bluebird bio Inc's BLUE study for patients under the age of 18 in studies evaluating lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD).
  • In December 2021, lovo-cel clinical studies were placed on a partial hold for patients under 18. The hold related to an investigation by bluebird bio into an adolescent patient with persistent, non-transfusion-dependent anemia following treatment with lovo-cel.
  • Results from a detailed investigation of this case were presented at the American Society of Hematology Annual Meeting, alongside details from another case of persistent anemia in an adult patient following treatment with lovo-cel. 
  • Related: FDA Okays bluebird bio's $3M Gene Therapy For Rare Neurological Disorder In Boys: What Investors Should Know.
  • Both patients had two α-globin gene deletions (−α3.7/−α3.7), also known as alpha-thalassemia trait, and notably are the only patients in the study with this specific genotype. 
  • Enrollment and dosing for adult patients 18 and older in the HGB-210 study continued as planned, while the partial hold was ongoing for patients under 18.
  • Bluebird remains on track to submit a Biologics License Application (BLA) to the FDA for lovo-cel in the first quarter of 2023.
  • As previously communicated, the company has completed the treatment of all patients in HGB-206 Group C, which will form the primary basis for efficacy in its lovo-cel BLA submission. It expects to complete vector and drug product analytical comparability studies for the lovo-cel BLA in Q4 of 2022.
  • Price Action: BLUE shares are down 6.49% at $7.19 on the last checked Monday.
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