Acting US FDA chief meets with rare disease groups to mend fences

NEW YORK — Acting U.S. Food and Drug Administration (FDA) Commissioner Kyle Diamantas met with rare disease advocacy groups on Wednesday as he works to strengthen relationships with a sector that had grown frustrated under the agency's previous leadership.

Representatives from organizations such as Friends of Cancer Research and the Foundation for Angelman Syndrome Therapeutics met with Diamantas to discuss the need for greater regulatory certainty and support for treatments targeting small patient populations.

The meeting comes as Diamantas seeks to stabilize the agency following the resignation of former FDA Commissioner Marty Makary last month. Makary's tenure drew criticism from some rare disease advocates after a series of regulatory decisions affecting drug developers in the field.

Among those decisions were the FDA's refusal to approve Biohaven's experimental treatment for a brain disorder, the addition of a serious safety warning to a Sarepta Therapeutics gene therapy, and comments from a senior agency official describing UniQure's Huntington's disease treatment as a "failed product."

Jeff Allen, chief executive officer of Friends of Cancer Research, welcomed the meeting and described it as a positive step toward improving communication between the FDA and the rare disease community.

"It was clear they are repairing the organization, and they are supporters of science and career staff," Allen said after the meeting.

A U.S. Department of Health and Human Services official confirmed the meeting, describing it as part of a broader effort to engage key stakeholders, including rare disease organizations.

Rare disease advocates have called for clearer regulatory guidance, noting that treatments for small patient populations often face unique challenges, including limited clinical data and difficulties in measuring safety and effectiveness.

According to attendees, FDA officials mainly listened to concerns raised by advocacy groups and made no specific commitments during the discussion. Participants emphasized the need for innovative clinical trial approaches, greater public engagement, and more predictable regulatory processes.

The Foundation for Angelman Syndrome Therapeutics also participated in the meeting and stressed the importance of regulatory clarity for the development of treatments for rare disorders.

Concerns about uncertainty at the FDA have been growing in recent months. In April, a coalition of rare disease organizations urged President Donald Trump and senior health officials to provide greater regulatory stability amid leadership changes at the agency.

Rare disease advocacy groups have previously found support from Trump. During his first term, he backed federal legislation allowing terminally ill patients access to experimental treatments, signing the measure into law in 2018.

Diamantas, who currently oversees the FDA's food division, is considered by some agency insiders and industry observers to be a steady leader despite lacking the traditional background often associated with the FDA's top position. Sources familiar with the process say he remains under consideration for a permanent appointment as commissioner. -Reporting by Chris Prentice and Michael Erman in New York and Yasmeen Abutaleb in Washington; Editing by Caroline Humer and Bill Berkrot/Reuters