Cure SMA Participates in a Special FDA Listening Session

Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order to gain a better understanding of the current goals, priorities, and challenges of our community.

Representatives from Cure SMA presented to the Commissioner and a panel of more than 25 key agency leadership, including Janet Woodcock, MD, Director, CDER; Billy Dunn, MD, Director, Division of Neurology Products, CDER; Jonathan Goldsmith, MD, Associate Director, Rare Diseases; Peter Marks, MD, PhD, Director, CBER; Diane Maloney, JD, Associate Director for Policy, CBER; Jeffrey Shuren, MD, JD, Director, CDRH; and, Gaytri Rao, MD, JD, Director, Office of Orphan Products Development.

Cure SMA provided the FDA with an overview of its top priorities and participated in a follow-up question-and-answer session with the agency’s top officials. Through open and clear dialogue, Cure SMA and the FDA will continue to work together to provide the most benefit to our community.

Representatives from Cure SMA presented on four key priorities for the SMA community:

  • Obtaining broad labels for drugs across SMA types, ages, and stages of disease progression.
  • Educating regulators on the severity of SMA on patients’ and families’ daily lives, including the importance of preventing decline or achieving small, incremental changes, and the importance of improving independence and activities of daily living.
  • Understanding the impact of the first approved drug on the design of future clinical trials, including the potential for multiple drug options and drug combination therapies.
  • The important need for and also the potential for flexibility on the use of placebo controls in SMA trials in the future.

The listening session, coordinated by FDA’s Office of Health and Constituent Affairs (OHCA), was moderated by Heidi Marchand, PharmD, Assistant Commissioner for Health and Constituent Affairs. In her opening remarks, Dr. Marchand emphasized the importance of these conversations so the agency can learn first-hand what the priorities and concerns are of FDA’s important stakeholders and determine what next steps are necessary to help address those issues.

“We were very grateful to have this important opportunity with Commissioner Califf,” said Kenneth Hobby, president of Cure SMA. “This is an exciting and important time for our community, with one drug currently being reviewed by the FDA and several others making substantial progress in clinical trials. With so much at stake, we realize the value in having one unified community—families, researchers, pharmaceutical companies, and regulators—working toward the same goals.”

The FDA Engagement Initiative

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 continue building strong relationships with the FDA and other regulatory bodies. We want to be sure the voice of our patient community is taken into account when the FDA considers these drugs for approval.

Participation in this FDA listening session is another facet of our ongoing FDA Engagement Initiative, which includes a number of activities designed to represent our community’s voice to the FDA. Use the links below to read more on our past work in this area:

You can also download the materials presented at this special listening session.

Do you like what you're reading?

Help make a difference in the lives of people affected by spinal muscular atrophy.

Leave a Comment

Your email address will not be published. Required fields are marked *

Scroll to Top