NVS : Data On Zolgensma Shows SMA Type 1 Patients Could Speak, Swallow & Maintain Airway Protection

(RTTNews) - Novartis (NVS) announced new data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy or SMA.

The completed Phase 3 SPR1NT study demonstrated that children with three copies of the SMN2 back-up gene who were treated presymptomatically achieved age-appropriate motor milestones, including standing and walking.

In addition, a descriptive post-hoc analyses of START, STR1VE-EU and STR1VE-US (n=65) indicated children with SMA Type 1 achieved or maintained important measures of bulbar function following treatment with Zolgensma, including ability to speak; swallow and meet nutritional needs; and maintain airway protection.

Bulbar motor neurons control muscles required for functions like swallowing, speaking and chewing, and impairment from SMA can lead to choking, malnutrition, infection and death.

Zolgensma (onasemnogene abeparvovec) is the approved gene therapy for the treatment of spinal muscular atrophy (SMA) and the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.

Zolgensma is now approved in more than 40 countries and more than 1,800 patients have been treated with Zolgensma globally across clinical trials, managed access programs, and in the commercial setting.