SMA Drug Pipeline Grows to 18 Programs

We’ve recently released an update to our SMA drug pipeline. This latest version shows the continued growth of the pipeline, setting several new marks for both breadth and depth:

  • We have 18 active programs.
  • We have 15 pharmaceutical partners.
  • We have seven programs in clinical trials.
  • The cumulative pipeline total is 26 programs, including eight failures to date.

All of these numbers are the highest we’ve reached in the 15-year history of the SMA drug pipeline. Cure SMA has provided funding for half of the ongoing drug programs.

Current Success, Future Challenges

The current pipeline reflects the success of our research model. As more and more of the drug programs we’ve already funded have had success, we’ve attracted more and more investment from pharmaceutical companies. Some of these pharmaceutical companies have been attracted to programs that we have provided seed funding for. And other companies, seeing the promise in the SMA research landscape, have taken the initiative to begin their own SMA drug programs.

We’ve already invested $57 million in SMA research, including over $25 million in the last 10 years. Because of the successes we’ve seen from these investments, and because of the unparalleled resources we’re able to offer in areas such as trial recruitment, we’ve already leveraged that into over $125 million in investment from pharmaceutical companies.

The results are impressive, but we still have more work to do. Only 10% of the drugs that make it to clinical trials will ultimately receive FDA approval, so we need to keep building and growing the pipeline. We also need to ensure that we are developing therapies that will be safe and effective for all types and all stages of SMA. Clinical trials and FDA approvals, and even insurance coverage are becoming urgent issues for our community. These are all due to our success in advancing the SMA drug pipeline.

Thank You

The dedicated support of our community has made all of this possible. Whether families raising funds and awareness, researchers investigating new potential treatments, or pharmaceutical and regulatory partners helping us take the next steps toward approval—everyone has a role to play. Thank you to everyone for their hard work and dedication.

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