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FDA proposes new treatment approval pathway for ultra-rare diseases

The Food and Drug Administration (FDA) on Monday announced a new proposal for flexible drug approval pathway treatments addressing ultra-rare diseases.

The FDA unveiled draft guidance on a proposed regulatory pathway for individualized therapies, treatments for rare conditions that affect a very small population, with the proposal specifically focusing on genome editing and RNA-based therapies. The FDA left open the potential for the framework to be applied to other treatments.

Developing these treatments is challenging due to the small number of patients who could feasibly take part in trials, limiting pretrial testing to support regulatory evaluation.

The proposed guidance, issued by the Center for Biologics Evaluation and Research, and Center for Drug Evaluation and Research, will apply to therapies targeting “a specific genetic, cellular or molecular abnormality.”

The framework lays out some key criteria for individualized therapies, including identifying the disease-causing abnormality; demonstrating that the therapy targets the root cause; and confirming successful target drugging or editing.

“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” Health and Human Services Secretary Robert F. Kennedy Jr. said in a statement.

“We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most,” he added.

Gene editing therapies have become a reality in recent years, with the FDA approving the first-ever CRISPR-based gene editing therapy in 2023, a treatment for sickle cell disease called Casgevy.

“My impression is that we are requiring the same level of evidence that we always have had for approval,” a senior FDA official said in a Monday press briefing.

“The idea with this pathway, or the plausible mechanism framework, is that it’s going to make the approval of these treatments, these very targeted treatments, more efficient, more rapid, and allow companies, when it’s appropriate, to enroll a smaller number of patients to generate the evidence for approval,” they added.

The official acknowledged this move was mindful of China’s growing dominance in the world clinical testing, including for gene therapies.

“We definitely want the United [States] … to lead in terms of innovation in, you know, the rare disease and the non rare disease space and we hope that this is going to contribute to that happening” said the official, though they added this single guidance proposal likely wouldn’t be enough for the U.S. to move ahead of China.

Copyright 2026 Nexstar Media Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

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