FDA taps Moderna’s mRNA therapy as sixth programme for rare disease pilot

2024-06-06

基因疗法临床研究临床申请信使RNAsiRNA

The FDA filled its second-to-last slot for a new rare disease accelerator with an mRNA therapy from Moderna, one of a dozen programmes that represent the biotech’s push to grow beyond its bread-and-butter of infectious diseases.

The candidate, mRNA-3705, is in the Phase I/II Landmark trial to treat methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase deficiency methylmalonic-CoA mutase deficiency in patients one year and older. The metabolic disorder, for which there are no approved therapies, is associated with significant mortality and can currently only be treated with a liver or combined liver and kidney transplants.

Kyle Holen, Moderna's head of development for its therapeutics and oncology unit, said that mRNA-2705’s inclusion in the pilot "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.” He added that the firm plans to start a pivotal trial of the candidate this year.

Boosting drug development

It’s the sixth experimental treatment to be announced for the FDA’s pilot programme, dubbed Support for Clinical Trials Advancing Rare Disease Therapeutics (START). Intended to speed up the development of mid- to late-stage drugs and biologics for rare diseases, participants can receive frequent advice from and communicate regularly with the agency’s staff on development issues such as study design and patient enrollment.

Launched in September of last year, the initiative is designed to ensure “promising treatments advance efficiently through regulatory milestones.”

Participating therapies will be split between drugs and biologics, with FDA’s Center for Drug Evaluation and Research in charge of selecting three products for rare neurodegenerative conditions, and the regulator’s Center for Biologics Evaluation and Research (CBER) responsible for picking four cell or gene therapies intended to treat a rare disease that is likely to lead to significant disability or death within the first decade of life.

The slate of candidates, which were first revealed at the end of May, includes Larimar Therapeutics’ nomlabofusp, a protein replacement therapy for Friedreich’s ataxia; Grace Science’s GS-100, an AAV9 gene therapy for NGLY1 deficiency NGLY1 deficiency; Myrtelle’s rAAV-Olig001-ASPA, a gene therapy for Canavan disease; Denali Therapeutics’ DNL126, an enzyme replacement therapy for MPS IIIA; and Neurogene’s NGN-401, a gene therapy for Rett syndrome.

One participant has yet to be announced.

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