FDA and C-Path Team Up over Rare Genetic Disease

The FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) are teaming up with the Critical Path Institute (C-Path) for a “pre-consortium” aimed at developing tools for use in clinical development programs for treatment of alpha-1 antitrypsin deficiency (ATTD), a rare genetic disease.

The condition puts individuals at risk of developing chronic obstructive pulmonary disease and other problems. ATTD also leads to progressive destruction of lung tissue leading to premature mortality or the need for lung transplant.  

One of the challenges in developing treatments is the need for biomarkers that would predict long-term outcomes.

C-Path is a non-profit group that includes more than 1,600 scientists from regulatory and research agencies, academia, patient organizations and bio-pharmaceutical companies. The pre-consortium aims to give the groups an opportunity to contribute to medical product development.

There is currently no cure for AATD and current treatments “do not adequately address the needs of patients,” the FDA said.