Empire State Review Team 
WASHINGTON, D.C. — In a significant policy shift aimed at bolstering the United States’ position in biotechnology innovation, U.S. Health and Human Services Secretary Robert F. Kennedy Jr. announced plans to expedite the approval process for treatments targeting rare diseases. The announcement was made during a Food and Drug Administration (FDA) meeting focused on cell and gene therapies held on June 5, 2025.
Kennedy emphasized the need to eliminate regulatory barriers that currently hinder the development and approval of therapies for rare conditions. “We are going to continue to figure out new ways of accelerating approvals for drugs and treatments that treat rare diseases, and we’re going to make this country the hub of biotechnology innovation,” he stated. The initiative aims to prevent companies from relocating clinical trials abroad due to existing regulatory challenges.
The proposal has garnered support from various stakeholders, including industry executives, researchers, and FDA officials. Vinay Prasad, the newly appointed head of the FDA’s Center for Biologics Evaluation and Research (CBER), committed to expediting access to promising therapies. Prasad, known for his critical stance on the FDA’s previous regulatory approaches, signaled a shift towards a more flexible and responsive framework for drug development.
The announcement had an immediate positive impact on the biotechnology sector. Shares of several therapy developers, including Sarepta Therapeutics, Dyne Therapeutics, Lexeo Therapeutics, and uniQure, experienced notable gains following the news.
The initiative also addresses concerns about the United States potentially falling behind other countries, such as China, in drug development. Panelists at the FDA meeting highlighted that the current regulatory hurdles may prompt companies to move clinical trials abroad or abandon them altogether. Carl June from the University of Pennsylvania remarked, “The path to approval is seen as so … . We cannot afford that exodus.”
The proposed changes come amid broader discussions about the FDA’s regulatory approach to rare diseases. Recent cases, such as the FDA’s rejection of Stealth BioTherapeutics’ treatment for Barth syndrome, an ultra-rare genetic disorder, have sparked debates about the need for a more accommodating approval process. Advocates argue that the current system may delay access to potentially life-saving treatments for patients with rare conditions.
Kennedy’s proposal represents a significant step towards addressing these concerns and ensuring that the United States remains at the forefront of biotechnology innovation. By streamlining the approval process for rare disease therapies, the initiative aims to foster a more conducive environment for drug development and improve access to treatments for patients in need.
