Clinical Trials Grants Program (RFA-FD-25-020)

Mitochondrial Disease Biomarker Development Program is a grant from the FDA Office of Orphan Products Development (OOPD) that funds clinical trials and natural history studies supporting the development of medical products for rare diseases, including mitochondrial conditions. The Orphan Products Grants Program, authorized by the Orphan Drug Act of 1983, awards grants to clinical investigators developing drugs, biologics, devices, and medical foods for conditions affecting fewer than 200,000 people in the United States. Eligible applicants include clinical investigators at academic institutions and research organizations. The program covers clinical trials evaluating efficacy and safety in support of new indications or labeling changes.

Summary: The fiscal year 2026 (FY26) Duchenne Muscular Dystrophy Research Program (DMDRP) Idea Development Award (IDA) promotes new ideas that are still in the early stages of development and have the potential to yield impactful data and new avenues of investigation. This award supports impactful, high-risk/high-reward research that could lead to critical discoveries or major advancements that will accelerate progress in improving outcomes for individuals with Duchenne muscular dystrophy (DMD) in the near term. Applications should include a well-formulated, testable hypothesis based on strong scientific rationale. The DMDRP strongly encourages research projects investigating therapies designed to demonstrate efficacy cross the life span, including infants, toddlers and nonambulatory individuals.Distinctive Features: The FY26 DMDRP IDA mechanism offers three eligibility career categories:• The Established Investigator category is for independent investigators at all academic levels, or equivalent• The New Investigator – Early-Stage category is for independent investigators early in their careers (i.e., within 10 years of their first faculty appointment or equivalent). Applicants in this category will be reviewed separately from Established Investigators.• The New Investigator – Transitioning category is for independent investigators at all academic levels, or equivalent, in an area other than muscular dystrophy who are seeking to transition to a career in DMD, thereby bringing their expertise to the field. Applicants in this category will be reviewed separately from Established Investigators.Preliminary data relevant to DMD that supports the feasibility of the research hypotheses and research approaches are required for all applications. Clinical trials or clinical trial aims are not allowed. Funding Opportunity Number: HT942526DMDRPIDA. Assistance Listing: 12.420. Funding Instrument: G. Category: ST. Award Amount: $2.5M total program funding.