Novartis' case for innovative gene therapy for spinal muscular atrophy gets stronger

Positive interim results from a Phase III trial evaluating the effect of Novartis’ (NOVN: VX) Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) in Type 1 spinal muscular atrophy (SMA) patients is expected to make the case stronger for its approval and also its long-term use as a mainstream treatment option, says GlobalData, a leading data and analytics company.

While the company waits in anticipation for a decision on the approval of the drug, the latest results are not surprising. So strong were the results from the pivotal Phase I START trial that it was used as the basis for filing the drug for approval in USA, Europe, and Japan. The interim results demonstrated prolonged event-free survival in Type 1 SMA patients who received the therapy as well as achievement of milestone developments compared to the natural history of the disease.

Vinie Varkey, senior neurology and ophthalmology analyst at GlobalData, comments: “Besides the strong results, this therapy’s mechanism of action is an attractive treatment as it is proposed to be administered as a one-time infusion compared to currently available Spinraza [nusinersen] from Biogen [Nasdaq: BIIB], which needs to be administered more frequently via an intrathecal administration; the latter represents a resource-intensive procedure and one that is considered risky for SMA patients who have undergone spinal fusions in order to address issues related to Scoliosis.”