The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp for the treatment of patients with Friedreich’s ataxia.
Friedreich’s ataxia is a progressive, inherited condition that affects the nervous system and causes movement problems. Common features include the gradual loss of strength and sensation in the arms and legs, spasticity, and impaired speech. Many individuals have a form of heart disease called hypertrophic cardiomyopathy. Some develop diabetes, impaired vision, hearing loss, or scoliosis. Most people with Friedreich ataxia begin to experience the signs and symptoms around puberty. This condition is caused by genetic changes in the FXN gene.
Nomlabofusp is a frataxin (FXN) protein replacement therapy being investigated for patients with Friedreich’s ataxia.
Following a Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program meeting with the FDA, there are plans to consider the use of skin FXN as a novel surrogate endpoint. This is likely to predict clinical benefit to support a Biologics License Application submission for accelerated approval planned for June 2026. An agreement was also made on the relevant clinical outcomes required for the nomlabofusp program.
BTD is designed to expedite the development and regulatory review of a drug intended to treat a serious condition. Eligibility is dependent on if preliminary clinical evidence indicates that the treatment may demonstrate substantial improvement over available treatments in one or more clinically significant endpoints.
The BTD and FDA feedback was based on the review of clinical data from an ongoing open-label study evaluating nomlabofusp in adult and pediatric patients with Friedreich’s ataxia. In this study, nomlabofusp was observed to increase skin FXN to levels expected in asymptomatic carriers and consistently improve four key clinical outcomes: modified Friedreich Ataxia Rating Scale (mFARS) score, FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) after 1-year on treatment.
Topline data from the open-label study and initiation screening in a global confirmatory phase 3 study are expected in Quarter 2 of 2026.
For more information, view the press release.
To learn more about Friedreich’s ataxia and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/