bluebird bio's Skysona at last gains FDA nod for rare brain disease

US biotech bluebird bio (Nasdaq: BLUE) has had a rocky ride in gaining US approval for its Skysona (elivaldogene autotemcel), but the US Food and Drug Administration (FDA), having earlier this year extended the review period for the product, on Friday granted authorization for the gene therapy.

Skysona, also known as eli-cel, is now cleared to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). The company also confirmed that the previous clinical hold on the eli-cel clinical development program has been lifted. Despite the positive news, bluebird’s shares closed down almost 5% at $6.50.

As a condition of the Skysona accelerated approval, bluebird has agreed to provide confirmatory long-term clinical data to the FDA. bluebird anticipates that this will include data from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years, and from commercially treated patients.