Larimar to seek nomlabofusp’s accelerated approval this year
Formerly CTI-1601, therapy is designed to deliver working frataxin to cells
Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to nomlabofusp, its experimental treatment for Friedreich’s ataxia (FA).
“The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA) submission expected by the end of 2025 to seek accelerated approval,” Carole Ben-Maimon, MD, president and CEO of Larimar, said in a company press release.
Accelerated approval is a type of conditional approval where marketing authorization can be granted based on early clinical data that suggests a treatment is likely to benefit patients. If accelerated approval is granted, Larimar would have to conduct additional testing to prove the therapy actually benefits patients.
Nomlabofusp, previously called CTI-1601, is designed to deliver a working version of the frataxin protein to patients’ cells. FA is caused by mutations in the gene that provides instructions to make frataxin, resulting in abnormally low levels of the protein.
Larimar intends to seek accelerated approval of nomlabofusp based on early data that suggests it leads to an increase in frataxin in patients’ skin cells. According to the company, available data suggest that if frataxin is increased in skin, it should also be increased in other tissues, including muscles, nerves, and the heart. Larimar said the FDA has signaled it’s open to considering skin frataxin levels as a basis for supporting accelerated approval.
“We are enthusiastic about the recent FDA interactions and their openness to consider skin frataxin (FXN) concentrations as a potential novel surrogate endpoint reasonably likely to predict clinical benefit in patients with Friedreich’s ataxia,” Ben-Maimon said. “Acceptability of [frataxin] as a novel surrogate endpoint will be based on review of the data by the [FDA] in the future BLA. As we previously disclosed, we have been collecting skin [frataxin] concentration data throughout the development program.”
Testing nomlabofusp
Larimar is testing nomlabofusp in a run-in study (NCT06681766), which is enrolling patients, ages 12-17, at a site in Maryland with the goal being to evaluate the therapy’s safety, tolerability and pharmacological profile. An open-label extension (NCT06447025) that’s testing its long-term effects in those who complete the run-in study along with adults from an earlier trial is also ongoing. Results are expected this summer.
Results presented last year showed sustained increases in skin frataxin levels, trends towards clinical improvement at three months, and positive tolerability data of daily injections under the skin, or subcutaneously, of 25 mg nomlabofusp for up to 260 days, or about 8.5 months. Also, dose escalation to 50 mg daily had already begun.
“Our open-label extension (OLE) study continues to advance, with some participants on daily treatment for up to one year [and the] run-in study continues to progress, and we expect to complete the dosing of the adolescent cohort by the end of this month and report available data in September 2025,” said Ben-Maimon, who noted the run-in study is expected to expand to include patients as young as 2.
Larimar is also gearing up for a Phase 3 clinical trial, which would be designed to definitively assess the clinical benefits of nomlabofusp in FA patients. The company has been communicating with the FDA and other regulatory agencies, and expects to launch the trial this year. Potential study sites are in the U.S., Europe, U.K., Canada, and Australia.
“We have received feedback from both FDA and European Medicines Agency (EMA) on the protocol for our global Phase 3 trial and are on track to initiate this trial mid-2025,” Ben-Maimon said.
About the Author
