FDA Licensing of Itvisma : Intrathecal Onasemnogene Abeparvovec
On the 24th November, Novartis pharmaceuticals have announced that the FDA have approved Itvisma® (onasemnogene abeparvovec-brve), a new version of the gene therapy, for treating children two years and older, teens and adults living with SMA.
Please read full press release from Novartis here
- Key Difference between Zolgensma and Itvisma®
- Zolgensma is administered intravenously (IV). It is funded by the NHS in the UK for children diagnosed with SMA Type 1 who weigh less than 21 kgs. It is also funded for babies who are pre-symptomatic and have up to 3 copies of the SMN2 gene.
- Itvisma is administered via a single intrathecal (IT) injection (directly into the spinal canal), allowing for a fixed dose that doesn’t need to be adjusted for age or body weight in older patients.
- Significance: This is the first and only gene replacement therapy approved for this broader, older population, offering the potential for a one-time treatment to address the root cause of SMA and possibly reduce the need for chronic therapies.
What Does This Mean for the UK?
- The Treatment Isn’t Available Here Yet
- Itvisma (intrathecal onasemnogene abeparvovec) does not currently have a license in the UK from the Medicines and Healthcare products Regulatory Agency (MHRA). It needs this license before it can be used by the NHS.
- The NICE Process Has Started – And Your Voice Matters!
- The National Institute for Health and Care Excellence (NICE) has already started the project to look at this treatment for people aged 2 years and over
- NICE needs to decide if a new drug works well and is good value for money for the NHS. This process relies heavily on hearing about the daily reality of life with SMA.
- SMA UK and our partner organisations will be working closely with the NICE appraisal committee to make sure the patient and carer perspective is clearly understood.
- Watch this space! As the process moves forward, SMA UK will be sharing opportunities for you to share your thoughts and experiences on the topic to inform the NICE committee.
SMA UK’s CEO Giles Lomax commented “We are delighted to see this announcement from Novartis today with Itvisma providing another treatment option for the broader spectrum of the SMA community. Over the next few months we will be supporting the NICE appraisal process which will involve engaging with the community and ensure we do everything we can to get access as quickly as possible in the UK.”
