FDA lifts hold on stud­ies of blue­bird's sick­le cell gene ther­a­py in ado­les­cents

In ad­vance of ask­ing for its third gene ther­a­py ap­proval, blue­bird bio said the FDA has lift­ed a clin­i­cal hold on its sick­le cell dis­ease can­di­date be­ing in­ves­ti­gat­ed in peo­ple un­der the age of 18.

The lift comes al­most a year to the day af­ter the Boston-area biotech dis­closed the reg­u­la­tor’s hes­i­tan­cy fol­low­ing a case of per­sis­tent ane­mia in an ado­les­cent, who was not de­pen­dent on trans­fu­sions.

Blue­bird said it re­mains on track to sub­mit a BLA for lovotibeglo­gene au­totem­cel (lo­vo-cel) in the first quar­ter of 2023. Al­so in that pe­ri­od, blue­bird will “re­sume en­roll­ment and treat­ment of pe­di­atric and ado­les­cent pa­tients,” chief med­ical of­fi­cer Richard Colvin said in a state­ment.

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