Cure SMA will be participating in a one-day workshop with The European Medicines Agency (EMA—the European equivalent of the FDA), SMA Europe and TREAT-NMD to discuss, support and advance the development of therapies for the treatment of SMA.

The workshop will convene 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 will include 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.

Kenneth Hobby, president of Cure SMA, will present on the patient perspective in drug development. The presentation will draw 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.

The workshop will take place at EMA’s premises in London on November 11, 2016. The entire day will be broadcast live and can be seen on the EMA’s website. Choose the “multimedia” tab for instructions on how to view the broadcast.

The Importance of Engaging With Regulators

When regulatory bodies such as the FDA or EMA evaluate a drug for approval, they must weigh many different factors, including the quantity and quality of evidence of safety and effectiveness, potential benefits of a treatment versus the potential risks, the impact the treatment will have on the patient community, and more. This is why it is so crucial that we build strong relationships with the FDA, EMA, and other regulatory bodies. We want to be sure the voice of our community is heard SMA drugs are considered for approval.

The EMA workshop is a key part of our ongoing work in this area, along with other activities such as our FDA Engagement Initiative, Hope on the Hill Congressional Dinner, and upcoming Patient-Focused Drug Development Meeting with the FDA.