Novartis' SMA gene therapy cleared for wider use in US

Novartis has a much-anticipated approval for its spinal muscular atrophy (SMA) treatment onasemnogene abeparvovec in the US, getting a green light for a new version that can make more patients eligible for the gene therapy.

The current Zolgensma version of onasemnogene abeparvovec is administered intravenously, which means it is only an option for younger children under a certain weight due to the risk of liver side effects, and is limited to use in the under-twos in the US.

The new Itvisma formulation is administered by an injection given directly into the cerebrospinal fluid through the lower back, making it an option for larger people living with SMA, including older children and adults.

The new intrathecal formulation means the one-shot gene therapy is now available to all SMA patients, regardless of their age, and could free them from having to take regular, lifelong SMA treatments like Biogen's Spinraza (nusinersen) and Roche's Evrysdi (risdiplam).

Novartis has previously guided "multibillion-dollar" sales potential for Itvisma at its peak.

Itvisma (formerly OAV101 IT) has been approved by the FDA on the strength of the phase 3 STEER study – which involved treatment-naïve patients with SMA aged from two to 17 who were able to sit, but had never walked independently – and the phase 3b STRENGTH trial in a similar group who had previously been treated with Spinraza or Evrysdi.

Itvisma will be launched next month in the US with a wholesale acquisition cost of $2.59 million, compared with $2.1 million for Zolgensma, according to Novartis.

The new version is expected to restore sales momentum for Novartis' SMA gene therapies, given that Zolgensma sales in the third quarter were slightly down from the prior year, at $301 million, as the pool of eligible patients starts to contract and it becomes an option mainly for newly diagnosed cases. Zolgensma has been available in the US market since 2019.

Patient advocacy group Cure SMA – which is due to hold a webinar for the SMA community on 11th December in association with Novartis to discuss Itvisma's approval – said it is "another critical step forward in the ongoing effort to treat and manage" the muscle-wasting disease.

"Cure SMA is committed to working closely with Novartis, healthcare providers, and the SMA community to support timely education and access to this new treatment, in alignment with its approved use," said the organisation.

Image by Steve Buissinne from Pixabay