European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for risdiplam for the proposed use in patients with spinal muscular atrophy (SMA).
Within the last 30 minutes we have received word that the EMA have accepted the submission from Roche for market authorisation to begin offering treatment with risdiplam across Europe. The following is the communication that we received from Roche just now:
“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.”
This is obviously great news but please be aware that this doesn’t mean the treatment is available widely in the UK, this is the first step in receiving approval. However, it is very good news that the compassion access provision has been extended. We will share more information with you soon as we have it.