Key facts
- Acting FDA Commissioner Kyle Diamantas met with rare disease organizations.
- The meeting aimed to repair relations after dissatisfaction with former Commissioner Marty Makary.
- Rare disease groups seek greater certainty and support for treatments for small patient populations.
- The FDA had previously rejected Biohaven's experimental treatment and warned on a Sarepta Therapeutics gene therapy.
- Diamantas is reportedly being considered for the permanent FDA Commissioner role.
Acting U.S. FDA Commissioner Kyle Diamantas met with representatives from rare disease organizations, including Friends of Cancer Research and the Foundation for Angelman Syndrome Therapeutics, on Wednesday. The meeting was part of an effort to repair relations with the rare disease sector, which had become dissatisfied with the agency under former Commissioner Marty Makary. Rare disease groups are advocating for increased certainty and support for treatments targeting small patient populations. During Makary's tenure, the FDA had issued setbacks for drugmakers in this space, including rejecting Biohaven's experimental treatment for a brain disorder and issuing a serious safety warning on a Sarepta Therapeutics gene therapy. Diamantas, who previously led the FDA's food division, is seen by some as a stabilizing figure and is reportedly being considered for the permanent commissioner role. A U.S. Department of Health and Human Services official confirmed the meeting, stating it was part of a broader initiative to engage with key stakeholders, particularly within the rare disease community.