On Monday 17 August, Roche announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for risdiplam for the proposed use in patients with spinal muscular atrophy (SMA). Risdiplam is an oral drug that increases SMN protein levels by targeting the SMN2 gene.
Validation of the MAA begins the formal scientific evaluation process by EMA’s Committee for Medicinal Products for Human Use (CHMP). The EMA has granted the marketing application with accelerated assessment, which means that the evaluation process can be reduced from 15 to 9 months, if the accelerated assessment timetable is maintained.
This is a significant step. As a result of the validation, Roche’s Compassionate Use Programme (CUP) has been extended to now include all Type 1 and Type 2 patients who have exhausted all therapeutic options, or Type 1 and Type 2 patients who have had their treatment interrupted as a direct result of the COVID-19 pandemic. In practice, this means that anyone with SMA Type 1 or 2 who is unable to receive Spinraza for clinical reasons, will be able to access risdiplam if their clinician thinks this is appropriate. Applications for compassionate use should be made to Roche by a treating clinician.
The CUP is available to people with SMA Type 1 or 2 right across the UK.
There is a full FAQs document from Roche on the CUP. These FAQs have been checked for accuracy with Roche where they relate specifically to risdiplam. Questions and answers apply to people living in England, Scotland, Northern Ireland and Wales.