EMA Validates Roche's Marketing Authorization Application for Risdiplam

17 August 2020

Roche UK Update:

In response to your request for information, please find the following update. I am pleased to let you know 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).

Validation of the MAA confirms that the submission is accepted and 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.

As the European Medicines Agency (EMA) has accepted the dossier submission for risdiplam, this has led to a planned compassionate use eligibility criteria adaptation in the UK, to include SMA Type 2 patients who have exhausted all therapeutic options.

The updated top-level eligibility criteria for risdiplam compassionate use provision is: 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. Applications for compassionate use should be made to Roche by a treating clinician. 

As soon as we have more information, we will pass it on to the Community.