FDA nod for Zolgensma in SMA; marks approval of most-expensive drug ever

The US Food and Drug Administration on Friday approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality.

The label for Zolgensma, a one-time treatment, has a less restrictive age limitation than analysts had anticipated. Importantly it will also allow Zolgensma to capture all Type 1 and Type 2 SMA patients without the need of newborn screening – Type 1 patients develop symptoms by 6 months of age, and Type 2 patients typically develop symptoms between the ages of 7 to 18 months, noted SVB Leerink Research analyst Mani Foroohar.

Following the FDA announcement, the gene therapy’s developer AveXis, a subsidiary of Swiss pharma giant Novartis (NOVN: VX) company, announced innovative access programs for Zolgensma for the treatment of pediatric patients less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.