RARE Daily

FDA Adds Moderna and Myrtelle to START program

June 6, 2024

Rare Daily Staff

The U.S. Food and Drug Administration selected Mytelle’s experimental gene therapy for Canavan disease and Moderna’s experimental mRNA therapy for methylmalonic acidemia as participants in its Support for Clinical Trials Advancing Rare Disease Therapeutics or START pilot program.

Neurogene’s NGN-401 gene therapy for Rett Syndrome, Grace Science’s GS-100 gene therapy for NGLY1 Deficiency, Denali Therapeutics’ enzyme replacement therapy DNL126 for the potential treatment of MPS IIIA (Sanfilippo syndrome type A), and Larimar Therapeutics’ experimental therapy nomlabofusp for Friedreich’s ataxia are participants the agency selected for the program.

The START pilot program was launched by the FDA in September 2023. It is intended to treat a rare disease or other serious condition with high unmet medical need through an enhanced mechanism for communication with the FDA.

Sponsors selected can benefit from more frequent and rapid ad-hoc interactions with the FDA to help facilitate the development of programs to the pivotal clinical study or pre-BLA meeting stage, and to generate high-quality and reliable data intended to support a BLA or New Drug Application.

Mytelle’s rAAV-Olig001-ASPA is a gene therapy in development to treat Canavan disease (CD). CD is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) that prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain bioenergetics and development, including myelin production. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD.

As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

Moderna’s mRNA-3705 is in development to treat methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. MMA is a rare, life-threatening, inherited metabolic disorder that is most commonly 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. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver or combined liver and kidney transplants are the only effective treatments.

“Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application,” said Nancy Barone Kribbs, senior vice president of global regulatory affairs at Myrtelle.

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