FDA Selects Moderna's Rare Disease Candidate For Accelerated Development Program

Thursday, the FDA selected Moderna Inc’s MRNA mRNA-3705 for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.

mRNA-3705 is an investigational therapeutic for methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency.

“Selection for this program will enable enhanced communication with the U.S. FDA, resulting in acceleration of our development program as we prepare for pivotal study initiation for mRNA-3705 in 2024,” said Kyle Holen, Moderna’s Senior Vice President and Head of Development, Therapeutics and Oncology.

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The FDA initiated the START pilot program 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.

Selected manufacturers will benefit from rapid, ad hoc FDA interactions that support clinical development, such as study design, patient population, and statistical methods, beyond standard formal meetings.

MMA is an inherited metabolic disorder most commonly (approximately 60% of cases) caused by a deficiency in the mitochondrial enzyme MUT.

This deficiency can lead to metabolic crises due to a toxic buildup of acids in the body, progressing into multi-organ disease.

Currently, liver or combined liver and kidney transplants are the only effective treatments.

mRNA-3705 is being investigated in a Phase 1/2 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 (hMUT) deficiency.

mRNA-3705 is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA and consists of mRNA encoding human MUT, the mitochondrial enzyme commonly deficient in MMA, encapsulated within Moderna’s proprietary lipid nanoparticle.

Price Action: MRNA shares are up 1.25% at $156.77 at last check Thursday.

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