Zolgensma data show rapid onset of response for SMA type 2
As Novartis awaits an FDA decision any day on its spinal muscular atrophy gene therapy Zolgensma, data presented over the weekend at AAN suggest that at least half of patients could see a benefit within the first month of treatment, giving it an edge over marketed SMA therapy Spinraza nusinersen.
The AveXis Inc. unit of Novartis AG (NYSE:NVS; SIX:NOVN) presented the first data for Zolgensma onasemnogene abeparvovec-xioi (AVXS-101) in patients with late-onset SMA known as SMA type 2 at the American Academy of Neurology meeting in Philadelphia on Sunday. Zolgensma, which the pharma gained via its $8.7 billion acquisition of AveXis a year ago, delivers the survival of motor neuron 1 telomeric (SMN1) gene using an adeno-associated viral 9 (AAV9) vector from RegenxBio Inc. (NASDAQ:RGNX)...