Roche announces global pre-approval access program (PAA, CUP) with Risdiplam

Good news came from Roche

The pre-access only applies to all SMA TYPE 1 patients of all ages. After approval, everyone with SMA type 2 has access, please read more at SMA Europe

Roche is currently offering patients with the most urgent medical need, Type 1 SMA, the opportunity for access to risdiplam based on their treating physician’s decision. Being mindful, however, that patients with other types of SMA are also facing life-endangering situations, the programme will be expanded to patients with Type 2 SMA at the moment of filing of the regulatory application for risdiplam in each country. This means that in participating countries within the European Union, the programme is already available for patients with Type 1 SMA and will expand to patients with Type 2 SMA upon filing of our Marketing Authorisation

Application (MAA) to the European Medicines Agency (EMA), which is currently planned for mid 2020.