Rare Disease: Closed Roundtable Shows the Hart and Growing Commitment from FDA

Research evidence excerpt

Rare Disease: Closed Roundtable Shows the Hart and Growing Commitment from FDA

Biotechnology

EQUITY RESEARCH Industry Report

June 4, 2026

Rare Disease: Closed Roundtable Shows the Hart and

Research Analysts:

Kristen Kluska Growing Commitment from FDA

212-915-1927

Kristen.Kluska@cantor.com What Happened?

Ayan Hussein, PhD

212-915-1122 ●The EveryLife Foundation for Rare Diseases (who Cantor had the

Ayan.Hussein@cantor.com pleasure of recently hosting during our Rare Disease Month Webinar

Ross Fladeland series) issued a press release with feedback from a closed roundtable 212-915-1803

ross.fladeland@cantor.com discussion with FDA leadership that took place yesterday (06/04)

(LINK to PR).

●It was a very thoughtful letter, and we recommend anyone who is in

the field or investing in it to read it.

What Stood Out To Us?

●It was Mr. Diamantas' first public meeting since he became Acting

Commissioner. Beyond this, several FDA leaders were there (including

Acting Directors of CBER and CDER), which is important, as we

recognize that many leadership positions may still change with recent

turnover.

●He emphasized his commitment to finding new approaches for rare

diseases, repeating that it is a top priority under this leadership.

●They also noted their assurance that personnel have resources,

authority, and support necessary for programs in this space.

●FDA intends to have more discussions and engagements with the

community. They also laid out a vision that included a science-first

approach, and to rely on the experts who form the backbone of FDA.

●Importance of FDA regulatory flexibility was stressed, including

tailoring approaches to individual circumstances, along with providing

greater consistency and transparency (without lowering scientific or

regulatory standards).