Nomlabofusp for Friedreich’s ataxia
Fact-checked by Joana Carvalho, PhDWhat is nomlabofusp for Friedreich’s ataxia?
Nomlabofusp, previously known as CTI-1601, is an experimental therapy being developed to ease the symptoms and potentially slow the progression of Friedreich’s ataxia (FA).
FA is caused by mutations in the FXN gene that result in very low levels of a protein called frataxin. This protein is important for the functioning of mitochondria, which are cellular structures involved in energy production. Low levels of frataxin disrupt mitochondrial function and leave cells vulnerable to damage, particularly nerve and muscle cells.
Nomlabofusp makes use of a carrier peptide to transport and deliver frataxin to mitochondria. By restoring frataxin levels, nomlabofusp is designed to improve mitochondrial function, ease FA symptoms, and potentially slow its progression.
Administered via an under-the-skin (subcutaneous) injection, nomlabofusp is being developed by Larimar Therapeutics. The therapy has previously received a series of designations in the U.S. and Europe meant to support its development and regulatory review, including orphan drug status, fast track, and rare pediatric disease designations.
While nomlabofusp is still undergoing Phase 3 testing, the submission of a rolling application to the U.S. Food and Drug Administration seeking its accelerated approval is underway.
Therapy snapshot
| Treatment name | Nomlabofusp |
| Administration | Subcutaneous injection |
| Clinical testing | Currently in Phase 3 testing; the submission of a rolling application seeking its accelerated approval in the U.S. is underway |
How will nomlabofusp be administered in FA?
In clinical trials involving people with FA, nomlabofusp was administered via subcutaneous injections at various doses and with different dosing schedules.
It is not yet known what the recommended nomlabofusp dosage and dosing frequency will be for FA if the therapy receives approval.
Nomlabofusp in FA clinical trials
Nomlabofusp was tested in several clinical trials involving people with FA:
- Earlier Phase 1 studies (NCT04176991 and NCT04519567) evaluated the safety and tolerability of single and multiple ascending doses of nomlabofusp. Results showed that the therapy was generally well tolerated and successfully increased frataxin levels in several tissues.
- A Phase 2 clinical trial (NCT05579691) enrolled 28 adults with FA to evaluate the safety and pharmacological properties of the therapy when administered at doses of 25 mg and 50 mg. Results showed that frataxin levels increased depending on the administered dose. After two weeks, participants who were given 50 mg doses had frataxin levels that were a third to half of those seen in healthy adults. Nomlabofusp was generally well tolerated.
- An ongoing open label Phase 2 study (NCT06447025) is enrolling FA patients ages 2 to 60, who have or have not previously participated in other trials of nomlabofusp, to evaluate the effects of treatment over a longer period. Adult participants received nomlabofusp at a daily dose of 50 mg, while younger patients were given weight-based doses. According to recent data announced by Larimar, in the 43 participants who had received nomlabofusp as of June 2026, frataxin levels increased and remained elevated with continued dosing. Measures of neurological function, ability to perform daily activities, motor function, and fatigue showed favorable trends among participants who were treated over the course of a year.
- The FORWARD-FA study (NCT07778836) is an ongoing Phase 3 clinical trial evaluating nomlabofusp in adolescents and adults with FA to confirm the therapy’s clinical benefits. Participants will be receiving daily treatment with nomlabofusp or a placebo for 72 weeks, with the main goal of determining whether treatment can improve the scores of motor function and balance measures.
Nomlabofusp side effects
As it remains an experimental therapy, nomlabofusp’s safety profile is still being investigated. The most commonly reported side effects in clinical trials have been mild or moderate reactions at the injection site, which have been less frequent with continued treatment.
Other reported side effects included:
- nausea
- hives or allergic reactions
- anaphylaxis, a potentially life-threatening allergic reaction
Friedreich's Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
