Moderna’s mRNA-3705 selected by FDA for START pilot program

Moderna announced that the U.S. Food and Drug Administration has selected mRNA-3705 for the Support for Clinical Trials Advancing Rare Disease Therapeutics pilot program. mRNA-3705 is an investigational therapeutic for methylmalonic acidemia due to methylmalonic-CoA mutase deficiency. The START pilot program was initiated by the U.S. FDA in September 2023 to accelerate the development of novel treatments addressing unmet medical needs in rare diseases, with an initial selection of up to seven novel treatments, three by the Center for Drug Evaluation and Research and four by the Center for Biologics Evaluation and Research. The milestone-driven initiative is intended to help the progression to pivotal clinical studies or pre-BLA/NDA meeting stages by enhancing communications between manufacturers and the U.S. FDA. Selected manufacturers will benefit from rapid, ad-hoc U.S. FDA interactions to support clinical development, such as study design, patient population, and statistical methods, beyond standard formal meetings. The program is designed to generate high-quality, reliable data to support marketing approvals, ensuring promising treatments advance efficiently through regulatory milestones. mRNA-3705 is being investigated in a Phase 1/2 study, called the “Landmark study,” an adaptive, open-label study designed to evaluate the safety and tolerability of the investigational therapeutic administered via intravenous infusion in patients one year and older with isolated MMA due to methylmalonyl-CoA mutase deficiency.