Cure SMA is pleased to announce the launch of Phase 2 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical to advancing drug development in SMA and will benefit the broader SMA community. The members of the SMA Industry Collaboration are Astellas, AveXis, Biogen, Genentech/Roche, Cytokinetics, and Ionis.

Collaborative industry groups like this one are a rare opportunity to advance goals important to the SMA community. We thank our industry partners for their example of “the strong sense of collaboration that is helping to bring…therapies for SMA to patients and families,” as Dr. Jonathan Goldsmith of the FDA recently remarked at the SMA Patient-Focused Drug Development Meeting.

In the first phase of the collaboration, the group coalesced around three major topics of importance to our community: engagement with the FDA and other regulatory agencies, education in the medical community to reduce time to diagnosis, and increasing clinical trial site capacity.

The signature accomplishment of this first phase was the execution of a successful Patient Focused Drug Development Meeting with the FDA, a project of the regulatory topic group.

(Read more about the Patient Focused Drug Development meeting here.)

In this second phase, the regulatory group will oversee the development of the Voice of the Patient Report as part of the follow-up to the PFDD meeting. The group will also work on plans for future engagement with the FDA.

In addition, the emphasis in this second phase will shift toward the other two topic groups, which have spent the first phase laying the foundation for their activities.

The education topic group will work on expanding awareness of SMA among families and caregivers as well as pediatricians and other healthcare providers, with a focus on reducing diagnostic delays. Diagnostic delays can put significant financial, logistical and emotional strain on families. More importantly, they can also reduce the opportunity for early treatment. The education topic group will work with stakeholders to improve recognition of SMA, to shorten the time to diagnosis and avoid unnecessary delays in beginning treatment.

The clinical trials group will focus on identifying and pursuing opportunities to increase site capacity and optimize readiness throughout the US. The goal is to improve our community’s access to clinical trial sites and increase capacity. In this phase, the topic group will focus both on assessing and improving existing clinical trial networks, as well as reaching out to potential new SMA clinical trial sites.

The activities of the SMA Industry Collaboration are jointly supported by Astellas, AveXis, Biogen, Genentech/Roche, Cytokinetics, and Ionis.