Novartis’ Zolgensma has first-line potential in spinal muscular atrophy but durability questions linger without long-term data, experts say

09 Jul 2019

Novartis’ (SIX:NOVN) Zolgensma (onasemnogene abeparvovec-xioi) has strong potential to be used as a first-line treatment in spinal muscular atrophy (SMA), but it is unclear how durable it will be despite being designed as a one-time treatment, experts said. Second-line options including Biogen’s (NASDAQ:BIBB) Spinraza (nusinersen) are being considered, but lack of clinical data attesting to post-Zolgensma use could cause payer resistance.

Analyst reports suggest strong uptake sentiment for IV Zolgensma due to its broad label, but some experts interviewed by this news service noted there are still unaddressed efficacy questions for Zolgensma, as pivotal data fails to account for older patients that fall within the label. While ongoing and upcoming trials seek to address some of those efficacy shortcomings, including use in older patients or patients with neutralizing antibodies, there is no guarantee of success in those trials.

This news service reported on 28 June that Novartis has plans for a 2020 Phase I trial in SMA infants who present neutralizing antibodies. At the recent American Academy of Neurology (AAN) annual meeting in May, Novartis announced positive interim data from its Phase I STRONG trial (NCT03381729) testing intrathecal Zolgensma delivery in older patients, and trial completion is expected in September 2020. The company also presented results from the STR1VE, SPRINT and STRONG trials at the recently concluded Cure SMA conference, although details have not been publicly released.

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