Novartis’ BLA accepted for gene replacement therapy

Editas and BlueRock to develop cell medicines
Credit: Sergey Nivens

The US FDA has this week accepted Novartis’ Biologics License Application (BLA) for AVXS-101, which will now be known as Zolgensma.

The treatment has also been granted Priority Review.

Zolgensma is an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1.

It previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019.

It represents the first in a proprietary platform to treat rare, monogenic diseases using gene replacement therapy – technology that replaces a missing or defective gene with a functional copy to correct the underlying cause of genetic disease