The FDA’s Accelerating Rare Disease Cures Program

by | Aug 3, 2023 | CDRH, FDA, Gene and Cell Therapies, Pharma, Pharmaceuticals, Quality, Regulatory

In May of 2022, the Center for Drug Evaluation and Research (CDER) at the Food and Drug administration (FDA) launched the Accelerating Rare Disease Cures (ARC) program. This program sought to bridge the complexities of rare disease drug development with the pressing needs of patients [1]. With this program’s initiation, the Rare Diseases Team was formed within CDER to work and maintain collaborative relationships between internal and external rare disease stakeholders and scientific experts [2].

As this program has now eclipsed its first year in existence, it has brought about a unifying force between the Center for Biologics Evaluation and Research (CBER), the Center for Devices and Radiological Health (CDRH), Oncology Center of Excellence (OCE), and, of course, CDER.  This has led to the optimization of the collaborative efforts from each of these FDA entities on both the research and regulatory sides. Ultimately, leading to more and higher quality research into these rare diseases.

The ARC program has helped to expedite the processes of research and FDA approval of drugs that can treat rare diseases that, otherwise, might have been overlooked due to their rare nature. In February of 2023, the FDA approved the first treatment for Friedreich’s ataxia which is a rare degenerative disease that damages the nervous system [3]. They also approved a treatment in April of 2023 for amyotrophic lateral sclerosis (ALS) which is associated with a mutation in the SOD1 gene [4].

If you need help understanding the regulatory pathways for the FDA to bring a medical device, drug, food, or supplement to market per FDA compliance, the team of experts at EMMA International can help! Contact us today at or by calling 248-987-4497.


[1] FDA. (2023, June 13). Accelerating Rare disease Cures (ARC) Program Emerges as a Conduit for Empowering Rare Disease Stakeholders. Food and Drug Administration.

[2] FDA. (2022, October 06). Rare Diseases Team. Food and Drug Administration.

[3] FDA. (2022, October 06). FDA approves first treatment for Friedreich’s ataxia. Food and Drug Administration.

[4] FDA. (2022, October 06). FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene. Food and Drug Administration.

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