Moderna Selected for FDA’s Rare Disease Therapeutics Pilot Program

First launched in September 2023, the START program aims to facilitate drug development for rare diseases by offering drugmakers consistent and ad-hoc communication with FDA staff to address specific development issues, such as trial design, control selection, and patient population considerations. Additionally, the program provides guidance on using non-clinical data to support candidates and product characterization.

To qualify for START, companies must be developing gene or cell therapies targeting neurodegenerative or genetic metabolic rare diseases likely to cause significant disability or death within the first decade of life. Moderna’s mRNA-3705, aimed at treating MMA, fits these criteria perfectly.

MMA is a rare, life-threatening metabolic disorder marked by the accumulation of acids and other toxic substances in the body, leading to metabolic crises and multi-organ damage. Diagnosed within the first few months of life, MMA carries a high mortality and morbidity burden, with no currently approved treatments available. Most cases of MMA are caused by a deficiency in the MUT enzyme, essential for breaking down amino acids and fatty acids.

Moderna’s mRNA-3705 targets the underlying disease pathway by delivering a MUT-encoding mRNA molecule encased in a proprietary lipid nanoparticle, designed for intravenous administration. The investigational therapy, which has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA, is currently being evaluated in a Phase I/II open-label study for patients aged one year and older.

Kyle Holen, head of development for therapeutics and oncology at Moderna, expressed the company’s excitement and pride over mRNA-3705’s selection for START. He noted that this would “enable enhanced communication with the U.S. FDA, resulting in acceleration of our development program.” Moderna is preparing for a pivotal MMA study for mRNA-3705 later this year.

“This selection highlights the promise of Moderna’s innovative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA,” Holen stated.

Moderna joins four other biopharma companies selected for START. Denali Therapeutics, Grace Science, Larimar Therapeutics, and Neurogene have been included in the program with their respective therapies for mucopolysaccharidosis type IIIA, NGLY1 deficiency, Friedrich’s ataxia, and Rett syndrome.

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