Draft Guide on Disseminated Valley Fever Treatments

FDA released a draft guidance to help drugmakers design clinical trials for treatments targeting disseminated coccidioidomycosis, also known as Valley Fever, a fungal disease caused by Coccidioides species. The document outlines the agency’s current thinking on trial designs, patient populations, and efficacy endpoints for evaluating potential therapies.

Coccidioidomycosis is endemic in parts of the southwestern United States, northern Mexico, and areas of Central and South America, according to the agency. While most cases involve mild or moderate lung infection, some patients develop severe disseminated disease that can affect the skin, bones, joints, and central nervous system, it says. Treatment often requires antifungal therapy lasting six months to a year or longer.

According to the guidance, early clinical studies should evaluate drug tolerability, pharmacokinetics, and preliminary efficacy, including when investigational therapies are used in combination with standard of care. FDA encourages sponsors to use clinically meaningful primary endpoints, such as improvements in symptoms or disease resolution, with secondary measures including serological and radiological data.

For pivotal Phase 3 programs, the agency generally expects two randomized, controlled trials but said a single robust trial with additional confirmatory evidence could also support approval. The guidance notes that superiority trial designs — such as comparing a new therapy plus standard of care against standard of care plus placebo — will often be appropriate, though alternative designs may be acceptable if discussed with regulators in advance.