- The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorizations for Bluebird bio Inc’s (NASDAQ: 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.
