FDA Green Light ;Novartis has secured FDA approval for Itvisma, an updated version of its gene therapy Zolgensma, extending treatment to older patients with spinal muscular atrophy (SMA).
More than six years after Zolgensma was first cleared for children under 2, Itvisma’s label now encompasses older individuals with a confirmed SMN1 gene mutation.
Like Zolgensma, Itvisma restores SMN protein production by providing a working copy of the SMN gene using an adeno-associated virus (AAV) vector. The active substance is the same in both products, but the mode of delivery is different: Itvisma is administered into the fluid around the spinal cord via an intrathecal injection, while Zolgensma is infused through a vein. This intrathecal formulation allows Itvisma to be used at higher concentrations in patients with greater body weight.
Novartis has set Itvisma’s wholesale acquisition cost at $2.59 million, a company spokesperson said. At this price, the one-time therapy is projected to be between 35% and 46% cheaper over a decade than currently available chronic SMA treatments, the spokesperson said.
Actual out-of-pocket expenses will differ by patient, but Novartis anticipates wide insurance coverage and has created a support program that, for eligible patients, could bring their costs down to actually nothing – $0.
Roughly 9,000 people in the U.S. are living with SMA, and most are older children, adolescents and adults, according to Tracey Dawson, head of U.S. neuroscience at Novartis.
Novartis, which is targeting a much broader SMA population with Itvisma (OAV101 IT), has estimated that the therapy could generate multibillion-dollar peak sales. Since its 2019 launch, predecessor gene therapy Zolgensma has been used to treat more than 5,000 patients worldwide and recorded $1.2 billion in revenue last year, making it the top-selling gene therapy globally.
Dawson said Itvisma is aimed at patients who may want to move away from the routine burden of chronic therapy. Novartis anticipates that many candidates for Itvisma will be people currently on Biogen’s Spinraza or Roche’s Evrysdi who are interested in a one-time option.
Itvisma did well as far as the switch population goes in the phase 3b Strength study. Among 27 patients who stopped treatment with Spinraza or Evrysdi before receiving Itvisma, motor function remained stable over 12 months based on the Hammersmith Functional Motor Scale Expanded (HFMSE). On the 66-point scale, patients experienced a 1.05-point increase in least squares HFMSE score one year after dosing with Itvisma.
Dawson said that although the figures may seem modest, the real-world effect for patients is substantial, such as being able to keep operating their wheelchair controls or maintain their ability to walk. Even the improvements that seem minor on the surface, like maintaining grip strength, are actually very important for the patients, she continued.
Impact of the FDA Green Light
With the FDA Green Light secured, Novartis plans to roll out its therapy globally, offering hope to thousands of patients suffering from debilitating muscle disorders. Experts predict that this approval will pave the way for further advancements in the treatment landscape, highlighting the importance of the FDA Green Light in bringing innovative solutions to those in need.
Novartis’ achievement of the FDA Green Light underscores its ongoing commitment to innovation in rare and complex conditions. By focusing on therapies that address unmet medical needs, the company ensures that breakthroughs like this reach the patients who need them most. The FDA Green Light approval also positions Novartis as a leader in the field of muscle disorder treatments.
With the FDA Green Light secured, Novartis plans to roll out its therapy globally, offering hope to thousands of patients suffering from debilitating muscle disorders. Experts predict that this approval will pave the way for further advancements in the treatment landscape, highlighting the importance of the FDA Green Light in bringing innovative solutions to those in need.
