Moderna, Myrtelle Selected for FDA Rare Disease Program

The FDA has launched the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) programme. This initiative, introduced in September 2023, is designed to accelerate the development of new drugs and biological products specifically targeting rare diseases. Through this programme, the FDA aims to provide sponsors with frequent advice and consistent communication to expedite the progression of their drug development programs.

Moderna and Myrtelle have both been selected to participate in this pilot programme. Moderna's entry involves the development of mRNA-3705, a potential treatment for methylmalonic acidemia (MMA) caused by methylmalonic-CoA mutase (MUT) deficiency. This investigational therapeutic is being evaluated in a Phase I/II Landmark study (NCT04899310), focusing on its safety and tolerability. The treatment is administered intravenously to patients aged one year and older who suffer from isolated MMA due to hMUT deficiency.

Myrtelle, on the other hand, is working on rAAV-Olig001, a gene therapy targeting Canavan disease (CD). CD is a severe genetic disorder characterized by the breakdown of white matter in the brain due to mutations in the aspartoacylase gene (ASPA). This mutation disrupts myelin formation, leading to a deficiency in the aspartoacylase enzyme. The therapy uses a novel vector from recombinant adeno-associated viruses (rAAVs) which specifically targets oligodendrocytes, the cells responsible for myelin production in the brain.

Myrtelle's rAAV-Olig001 is currently undergoing a Phase I/II clinical trial (NCT04833907) involving 24 patients with CD. The treatment is administered directly to affected brain regions through neurosurgical procedures. This open-label clinical trial includes longitudinal clinical assessments and brain imaging as outcome measures.

Nancy Barone Kribbs, Myrtelle's Senior Vice President of Global Regulatory Affairs, emphasized the importance of the START programme by stating that the initiative allows for more direct communication with the FDA. This facilitates quicker resolution of development challenges that could otherwise delay market application.

Additionally, the pilot programme includes other notable participants. The Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) have selected a total of seven participants, with four chosen by CBER and three by CDER. Among these are Denali Therapeutics, Neurogene, Larimar Therapeutics, and Grace Science. Notably, Neurogene is investigating NGN-401, a gene therapy for Rett syndrome.

The START programme represents a significant step towards addressing the unique challenges of rare disease drug development. By providing a structured framework for more efficient communication and support, the FDA aims to streamline the journey from research to market for novel therapies, ultimately benefiting patients with rare diseases who have limited treatment options.