Cure SMA to Meet with FDA for Critical Path Innovation Meeting

We are excited to share with the SMA community that the U.S. Food and Drug Administration (FDA) has granted Cure SMA a Critical Path Innovation Meeting (CPIM). This means that key leaders at the FDA will have the opportunity to communicate with clinical investigators, members of academia, industry, scientific groups, and patients to improve efficiency and success in the next stages of drug development for SMA. At the upcoming virtual CPIM on August 4, Cure SMA seeks to enhance the understanding among these regulatory leaders of the most significant unaddressed needs for treating children and adults with SMA.

With multiple FDA-approved treatments for SMA, Cure SMA will discuss the community’s ongoing medical needs. The goals of this 90-minute CPIM will add to those of the successfully held Patient-Focused Drug Development Meeting (PFDD) for SMA with the FDA in 2017. At the PFDD meeting, more than 400 families, affected adults, and other SMA stakeholders came together to share what mattered most to the SMA community and  expectations for treatment.

Cure SMA’s discussion with FDA on August 4th will center around the effects of treatment on the quality of life of affected individuals, parents, and caregivers with SMA, as well as the tangible ways that having access to treatment has positively impacted the SMA trajectory for many of the 10,000+ Americans living with SMA. However, Cure SMA will also discuss the continued impact of SMA in our community following the approvals of treatments. This includes the distinct trajectories of affected children and adults who are treated after significant symptom development, compared to those treated earlier in disease progression.

Finally, we hope to discuss opportunities to collaborate with the FDA to help address existing unmet medical needs. As we continue to advance a new line of therapies for SMA, we must address new scientific and regulatory issues, including the following topics:

  • The changing natural history of SMA and continued, unmet medical needs among older patients with chronically progressing disease (i.e., people with SMA Types 2 and 3).
  • Development of novel outcomes in SMA, particularly for adults with SMA.
  • Exploration of novel biomarkers—specifically, the already identified neurofilament (NF) as a marker of disease progression in clinical trial and real-world settings.
  • Approaches to a combination of therapies as new therapeutic candidates in development, mainly those with complementary mechanisms of action to those already approved, will be tested in clinical trials and used in an additive fashion in the real-world.

Cure SMA representatives, as well as key experts in SMA basic and clinical research, clinicians (doctors and physical therapists), and industry partners will participate in this discussion. Two patient representatives, a mother to two boys with SMA Type 1 and an adult with SMA, will share their journeys as part of this discussion.

An action plan of agreed-upon priorities between Cure SMA and the FDA will result from this meeting. This may affect the design of future clinical trials, expectations for future therapies, and ultimately help to better meet the changing needs of our community.

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