The US Food and Drug Administration (FDA) is collaborating with the Critical Path Institute (C-Path) to organize a consortium dedicated to developing clinical tools for studying alpha-1 antitrypsin deficiency (AATD) treatments.

“There’s an urgent need for biomarkers in AATD that would predict long-term outcomes to use as surrogate endpoints for clinical trials of new therapies as well as a need for biomarkers that could be used to support efficient drug development, such as biomarkers for enrollment,” the FDA wrote on its official website.

The collaborative preconsortium invites knowledge sharing between industry, regulatory agencies, academia, and the patient community to find solutions for accelerating medical product development and ultimately improve care to those living with AATD and their families.

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The partnership involves 2 FDA centers, the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), and C-Path, an independent, nonprofit, public–private organization dedicated to the optimization of the medical product development process. C-Path creates new data, measurement, and methods standards to help researchers evaluating the effectiveness and safety of new therapies.

Read about AATD treatment

In the press release, the FDA reiterated its commitment to help find solutions for the challenges in rare disease drug development by working in close association with advocacy groups, academics, industry, patients, and other knowledgeable partners.

AATD is an inherited, genetic, and potentially life-threatening condition with no cure currently available. The search for new treatments has been compromised by the lack of appropriate tools for use in clinical development programs. Consequently, no new treatments have been developed over the last 3 decades and the existing ones do not fully address patient needs. Therefore, the search for new therapeutic options for AATD is essential.


FDA launches AATD pre-consortium partnership with the Critical Path Institute. News release. US Food and Drug Administration; June 10, 2022.