The European Medicines Agency (EMA) has recommended granting a marketing authorization in the European Union (EU) for Spinraza. The recommendation indicates that Spinraza should be covered for all types of SMA.
This recommendation is the first step in a three-step process to bring Spinraza to European patients. Next, the EMA’s recommendation will go to the European Commission for review and adoption. Once a marketing authorization has been granted, each EU Member State will issue a decision about price, reimbursement, and how Spinraza will be used in their respective country’s national health system.
In recommending Spinraza for marketing authorization, the EMA cited the significant unmet medical need in SMA, as well as the positive data from the clinical trials conducted by Biogen and Ionis.
Further, the EMA also cited the impact of their November 2016 workshop held with Cure SMA, TREAT-NMD and SMA Europe.
The workshop convened key stakeholders—patients, doctors, industry representatives, researchers and regulators—to take stock of the latest scientific developments in the area and discuss ways of developing therapies for the treatment of SMA. Topics for discussion included an overview of the disease, the pharmacology of the molecules under investigation, natural history data, clinical outcome measures and potential use of biomarkers in drug development.
Cure SMA presented on the patient perspective in drug development. The presentation drew from the community viewpoints we have collected from focus groups, surveys, and the Voices of SMA project. Cure SMA also provided funding support for this meeting.