Biogen announced today that the European Commission (EC) has granted a marketing authorization for Spinraza for the treatment of spinal muscular atrophy (SMA), making it the first approved treatment in the European Union (EU) for SMA. Spinraza was reviewed under the European Medicines Agency’s (EMA) accelerated assessment program, intended to expedite access to patients with unmet medical needs.

“Today we join individuals and families affected by SMA across Europe in celebrating the approval of Spinraza. Based on the robust efficacy and safety profile demonstrated in the clinical trials, we believe Spinraza will have a meaningful impact on infants, children and adults living with this devastating disease,” said Michel Vounatsos, chief executive officer at Biogen. “As part of our mission to improve the lives of those affected by SMA, we remain steadfast in our commitment to work with healthcare professionals, advocacy groups and government agencies to ensure people who could benefit from Spinraza receive access to this important treatment as quickly as possible.”

This authorization is the second step in a three-step process to bring Spinraza to European patients. In the final step, 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 April, when the EMA first recommended granting this marketing authorization for Spinraza, 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.

Read Biogen’s EU community statement on the granted marketing authorization for Spinraza.