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Moderna's MMA candidate nabs spot in FDA pilot for rare disease drugs

2024-06-07

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Methylmalonic acidemia, or MMA, is a genetic liver condition that in most cases is caused by a deficiency in an enzyme called MUT.

Methylmalonic acidemiac acidemia candidate, mRNA-3705, will be developed under the FDA’s START pilot program for rare disease, the company announced June 6.

Modernas tmethylmalonic acidemiaherapeutics,mRNA-3705e, Grace Sciences and Larimar FDArapeutics in the initiative, which aims to do for rare disease drugs what Operation Warp Speed did for the COVID-19 vaccines.

Methylmalonic acidemia, Denali Therapeuticsc NeurogenediGrace Sciencesost cLarimar Therapeuticsdeficiency in an enzyme called MUT, which processes fatty acids. Without it, the acids build up anCOVID-19 vaccines COVID-19 organs. The disease is usually diagnosed within the first few months of life, and there are currently no treatments.

Methylmalonic acidemiaased drug that encodes MUT, thereby restoring acid metabolism. It has orphan-drug, fast-track and rare pMUTatric disease designations from the FDA and is currently being tested in a 63-subject, phase 1/2 clinical trial.

MRNA-3705RT, mRNA-2705 will get extra attention from FDA officials, who will guide Moderna through product development issues like designing studies, choosing controFDAroups and selecting the right patient population. The effort is being carried out by the agency’s Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research, which is selecting six participants total for the first iteration of the program, according to a September 2023 announcement from the FDA.

“This selection highlights the promise of Moderna’s iFDAvative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA,” Kyle Holen, M.D., senior vice president and head of development in Center for Biologics Evaluation and Researcha, said iCenter for Drug Evaluation and Researchprogram will…[result] in acceleration of our development program as we prepare for pivotal study initiation for mRNA-3705 in 2024.”FDA

Moderna's other rare disease programs inclModernags for propionic acidemia, glycogen storage disorder type 1a, ornithine transcarbamylase deficiency, phenylketonuria, Crigler-Najjar syndrome type 1 and cystic fibrosis.Moderna mRNA-3705

Modernacked for the START program is Calico Life Sciencepropionic acidemia fglycogen storage disorder type 1aloornithine transcarbamylase deficiency ornithine transcarbamylaseMatter diseasphenylketonurian Crigler-Najjar syndrome type 1e bracystic fibrosiser caused by variations in an essential enzyme in the cells called eIF2B. This causes a reduction in enzymatic activity that can lead to chronic activation of the integrated stress response. This causes the white matter to degenerate, causing symptoms such as impaired muscle movement, cognitive decline and seizures. Patients have a shortened life span.