Today marks another landmark day for the adult SMA community in Australia, with the positive recommendation of Evrysdi (Risdiplam) for those in the community over 19 years of age. The positive recommendation from the PBAC is a testament to the effectiveness and safety of the drug and represents a significant step forward in the available options for those adults in the SMA community. With this new option available patient will be able to see improved outcomes and better quality of life.
We still have some work to do in order to get a listing date, but we will continue to work with the relevant stakeholders to ensure a timely listing date in the near future.
We would like to take this opportunity to those people who helped us gather the evidence needed (over and over) to show the unmet need for the adult community. The result is for you all to enjoy today.
If you are reading this and you are not connected to a doctor, please reach out to us at email@example.com and we will endeavour to get you connected ASAP before a listing date so you can start treatment promptly when Evrysdi is listed.
This announcement is a double celebration, with the listing for pre-symptomatic patients with 1 and 2 copies of SMN2. This is also a particularly important step forward in the treatment of those who are picked up via the newborn screening program that is rolling out for SMA around the country. This will make sure that those who are detected have timely access to the treatment they choose at birth. This is a phenomenal result and will have better outcomes and quality of life. We have already started to see new generation SMA that does not follow the path of what SMA used to look like. These outcomes can only happen due to successful listings like these.