2016 Update on SMA-FDA Interactions

The SMA drug pipeline has grown dramatically in just over a decade. Of the 18 programs in the pipeline, six of these are in clinical trials, and several of those are in Phase 2 and Phase 3 clinical trials.

Reaching and achieving FDA approval is a major step in our path to a treatment and cure. And, consistent with our strategy of pursuing many different therapeutic avenues, we may face this step many times.

When the FDA evaluates 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 and other regulatory bodies. We want to be sure the voice of our patient community is heard when the FDA considers these drugs for approval.

The Cure SMA FDA Engagement Initiative

In continuation of Cure SMA’s FDA Engagement Initiative, representatives have met regularly with FDA and other patient stakeholder groups to discuss patient perspectives and the importance of patient input in the drug development process. These meetings have been possible in part due to FDA’s work on the Prescription Drug User Fee Act (PDUFA) and the Patient-Focused Drug Development Initiative (PFDD).


Every five years, Congress must renew the Prescription Drug User Fee Act (PDUFA), which is the law that allows FDA to collect fees from drug manufacturers to fund the new drug approval process and other related activities. When PDUFA was reauthorized in 2012, known as PDUFA V, a stronger emphasis was placed on patient input in the drug development process. This resulted in the creation of the Patient-Focused Drug Development (PFDD) Initiative, to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition.

As part of this commitment, FDA pledged to hold at least 20 public meetings over the course of PDUFA V, each focused on a specific disease area, as well as offering participation in other community-wide meetings.

Voices of SMA

Late last year, Cure SMA set out to create a personal and effective tool with which to educate FDA staff as part of the PFDD Initiative. Through dozens of stories from the SMA community, Cure SMA created The Voices of SMA booklet, which described SMA drug development priorities, the impact of SMA on patients and their families’ daily lives, potential progress for patients, and our preferences and expectation for improved treatment.

The booklets, which were enthusiastically received by members of FDA leadership will be used to inform the agency during the review process when an SMA drug is brought forward for approval. In response to the Voices of SMA, Janet Woodcock, MD, Director of the FDA’s Center for Drug Evaluation and Research, shared, “Thank you. This is very moving and informative. We have to salute the courage of the families dealing with this terrible condition—they are continuing to fight! We have heard from other patient groups, who have serious debilitating illnesses, that improvements that would be ‘small’ in a healthy person can be extremely meaningful if one’s function is severely compromised.”

PDUFA Reauthorization

As we are nearing the end of PDUFA V, Cure SMA began working with FDA and other stakeholder groups at the end of 2015 to work towards reauthorization of the law that is set to expire in September 2017. As part of the process to reauthorize this law, the FDA held monthly meetings with stakeholders, patient groups, health care professionals, and academic experts to discuss ways to improve the drug review process. These meetings were concluded in March and were used to inform the agreement between FDA and industry that will be sent to Congress in the Fall of 2016 for reauthorization next year.

More About Our Recent Activities

  • PDUFA Reauthorization: Cure SMA actively participated in all 6 PDUFA reauthorization meetings held by FDA. Representatives provided testimony before FDA at the December meeting highlighting the importance of patient centered drug development and subsequently submitted comments that reiterated the SMA community’s priorities of incorporating the patient voice throughout all stages of drug development and approval.
  • Voices of SMA was created thanks to the success of the 2015 August Awareness month, which generated unprecedented levels of public awareness and outreach for SMA. As a result, the community seized on this momentum and turned awareness into action by helping Cure SMA develop the collection of patient stories, which were meaningful tools to carry SMA patients’ message directly to FDA leaders and Congressional representatives about the vital importance of considering the patient perspective in their deliberations and decision making.
  • On December 1st, 2015, we hosted our 5th Annual “Hope on the Hill” Congressional Dinner. Several Members of Congress attended the dinner, including Speaker of the House Paul Ryan, Congressman Sean Duffy, Congressman Richard Hudson, Congressman Bill Huizenga, Congressman Erik Paulsen, Congressman David Schweikert, and Congressman Greg Walden. The evening included remarks from Speaker Ryan, Congressman Walden (OR), and Cure SMA Board Member Greg Zerzan.

The 2016 Annual SMA Conference

At the 2016 Annual SMA Conference, we are hosting two thought-provoking and informative panels that bring researchers and families together: a moderated panel discussion on the new drug application (NDA) process, and a panel on SMA drugs in clinical development.

For More Information:

The Voices of SMA Booklet

5th Annual “Hope on the Hill” Congressional Dinner Recap

FDA/PDUFA Testimony

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