Bluebird bio's Neurological Disorder Gene Therapy Wins CHMP Backing for Approval

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorizations for Bluebird bio Inc’s BLUE Skysona (elivaldogene autotemcel) new gene therapy for children with a rare, inherited neurological disease.
The gene therapy is for those younger than 18 who have what’s known as early Cerebral Adrenoleukodystrophy (CALD).
EMA’s recommendation is based on a single-arm trial in 32 boys with CALD who received the one-time treatment, compared to 59 boys who had a stem cell transplantation.
“An analysis conducted after 24 months from the infusion on 30 subjects enrolled in the study concluded that for 27 of them (90%), treatment with Skysona preserved motor function and communication ability and improved survival when compared to untreated patients at an early stage of cerebral disease,” the EMA said.
Bluebird bio said it is currently on track to submit the marketing application to the FDA by mid-2021.
Price Action: BLUE shares are up 1.89% at $31.30 on the last check Friday.