Queries regarding pre-approval access/ compassionate use (PAA/CU) plans for Risdiplam
On 14 January 2020 Roche announced a pre-approval access/ compassionate use programme for its investigational drug Risdiplam to their affiliates globally and to patient advocate groups in Europe and UK.
According to the community letter shared by SMA Europe this program will initially be for Type 1 SMA but will expand to Type 2 SMA upon filing for market authorisation.
To our knowledge, there is no communication (yet) to Asia. We will monitor this together with other countries in our Asia Pacific - South Asia SMA network.
I am communicating with Roche Global SMA Team and they guided us to our local Roche affiliate, who we are in regular communication with. We are scheduled to catch up again in the next few days and will quickly send an update once additional information and guidance relevant to Malaysia is ready to be shared.
If you have any questions now, please fill out this form.
This is a positive and hopeful start to 2020!
Edmund Lim, Executive Director, Persatuan WeCareJourney