Nomlabofusp for Friedreich’s Ataxia
Discovered by R. Mark Payne, MD, at Indiana University School of Medicine, nomlabofusp is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA). Based on the results of our nonclinical development program, we believe nomlabofusp is processed to mature frataxin and becomes active in mitochondrial metabolism. Due to a genetic abnormality, patients with FA are unable to produce enough of this essential protein. Currently in a Phase 2 trial, nomlabofusp has been granted Rare Pediatric Disease designation, Fast Track designation, orphan drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and a PRIME designation by the EMA. Larimar’s nomlabofusp development program was also selected by the FDA to participate in its START (Support for clinical Trials Advancing Rare disease Therapeutics) pilot program, which is designed to accelerate development of novel therapies intended to address an unmet medical need for rare diseases.
About Friedreich’s Ataxia (FA)
Friedreich’s ataxia (FA) is a rare, progressive, multi-symptom genetic disease that typically presents in mid-childhood and affects the functioning of multiple organs and systems. The most common inherited ataxia, FA is a debilitating neurodegenerative disease resulting in multiple symptoms including progressive neurologic and cardiac dysfunction – poor coordination of legs and arms, progressive loss of the ability to walk, generalized weakness, loss of sensation, scoliosis, diabetes and cardiomyopathy as well as impaired vision, hearing and speech. FA affects an estimated 20,000 individuals globally. Approximately 5,000 of those affected with FA live in the United States and the majority of the remainder reside in Europe. FA results from a deficiency of the mitochondrial protein, frataxin (FXN), which is found in cells throughout the body.
We are currently studying nomlabofusp, a recombinant fusion protein intended to deliver human frataxin to patients with Friedreich’s ataxia in a Phase 2 trial. For more information, click here.
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