Cure SMA Plans a Series of Educational Modules for the SMA Community

With the recent news that Biogen and Ionis will submit nusinersen for FDA approval—along with other recent developments, including the breakthrough designation for AveXis’s gene therapy program, and ongoing work with other clinical trials—we know that many in our community have questions.

We have reached a new stage in our push for an FDA-approved treatment for SMA, and Cure SMA is committed to making sure our families, friends, and supporters have all the necessary information to understand and participate appropriately in the coming months.

To that end, we have planned a series of educational modules to address the topics, issues and questions that will be most critical as we move forward. All modules will include an interactive learning opportunity, such as a webinar, as well as booklets, handouts, and other materials that our community can review and refer back to. Webinars will be recorded and posted online for those who are not able to participate during the live session.

Please watch our website and social media channels for further announcements on the specific dates and times for each of the following modules.

New Drug Applications (NDA) and Expanded Access Programs (EAPs)

This module covers what our community can expect as nusinersen and future drugs move through the FDA’s approval process, and what provisions are made for access to the drug while that process is ongoing.

Patient-Focused Drug Development and Opportunities for Advocacy with the FDA

As part of the Prescription Drug User Fee Act (PDUFA), the FDA is required to take the patient voice into account when considering drugs for approval. This module will explain this patient-focused drug development initiative, and outline ways our community can participate.

The SMA Community and Industry Relationships

This module addresses how the community and Cure SMA are working with the pharmaceutical companies developing SMA drugs—before, during, and after the approval process.

Insurance and Coverage

This module covers the important topics of insurance and coverage for SMA drugs. How do we ensure that, once approved, drugs are broadly covered for those affected by SMA?

FDA Advisory Committees

For approval of the first treatment for a rare disease like SMA, the FDA is likely to convene an Advisory Committee (often called an “ad comm”) as part of their review. This module will discuss the ad comm process, how to participate, and what our community can expect at this stage.

Post-Approval Issues

This module will cover topics and questions about what happens after a first drug is approved for SMA. This includes the impact on the standard of care, what will happen with subsequent drug approvals, combination therapies, and more.

For More Information

For more information on how Cure SMA is working on the clinical and regulatory issues that are important to our community, please see these recent articles from our news section.

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