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# Guide on Developing Drugs for Lyme Disease
- URL: https://www.fdaweb.com/guide-on-developing-drugs-for-lyme-disease/
- Published: 2025-06-25T12:00:00.000Z
- Updated: 2026-09-14T15:15:13.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5159417

FDA has released a [final guidance](https://www.fda.gov/media/164949/download?ref=fdaweb.com) entitled “Early Lyme Disease as Manifested by Erythema Migrans: Developing Drugs for Treatment.” The document provides FDA’s current recommendations regarding the development of drugs to support an indication for the treatment of early Lyme disease as manifested by erythema migrans (EM).

The guidance says trials should enroll participants with early localized (i.e., a single EM lesion) or early disseminated (i.e., multiple EM lesions) disease, who reside in or have traveled to a Lyme-endemic area. “In general, sponsors should not enroll participants with musculoskeletal, neurologic, or cardiac manifestations of Lyme disease (e.g., active arthritis, myocarditis, meningitis, cranial neuropathy),” it says. Also, the agency says sponsors should not enroll participants with ongoing symptoms attributed to a history of Lyme disease or a concurrent tick-borne infection (e.g., babesiosis, ehrlichiosis, anaplasmosis).

Regarding trial designs, the guidance says trials are expected to be randomized and controlled because of the challenge with making causal inferences for the disease in a nonrandomized setting with potential differences between treatment groups in baseline disease characteristics such as time from infection, degree of dissemination, or symptoms. “Trials are also expected to be double-blinded due to potentially subjective elements in recommended endpoints and the unknown extent of possible open-label biases that could affect patient management and follow-up,” it says. It notes that superiority trials with a direct comparison to an approved drug or as an add-on design are acceptable to support an indication of treatment for early Lyme disease.