Roche Announces Global Compassionate Use Programme for Risdiplam

13 January 2020

This very positive development enables those who have SMA Type 1 global access to this promising treatment with plans to expand access to those with SMA Type 2 later in the year.

Currently in the UK, there is no structured pre-approval access programme. Roche hopes later in the year to have an Early Access to Medicines Scheme (EAMS) application to the UK Medicines and Healthcare Products Regulatory Agency (MHRA) approved.  

In the absence of any pre-approval access programme, patients may speak to their clinician about an application to Roche UK for compassionate use. Applications will be considered on a case by case basis in line with the eligibility criteria set out for the global programme in the global team’s community letter and Roche’s position paper on access to investigational medicines.

Roche UK has provided more detail on the global programme and how it relates to the UK. You can read this here.

Further information

Roche Global Team Programme announcement

Roche UK's statement

Roche’s Position Paper

More about risdiplam