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Nomlabofusp · 1 trial · 2 indications
Number - The USS has a score range from 0 to 36. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Number - The mFARS has a total score range from 0 to 93. Lower scores mean less impairment and higher scores mean greater neurological impairment.
| Arm | Type | Description |
|---|---|---|
| Nomlabofusp | EXPERIMENTAL | Daily subcutaneous injection of nomlabofusp for 72 weeks |
| Placebo | PLACEBO_COMPARATOR | Daily subcutaneous injection of placebo for 72 weeks |
| Name | Type | Description |
|---|---|---|
| Nomlabofusp | DRUG | Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia. |
| Placebo | DRUG | The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection. |
Key Inclusion Criteria: Subjects who meet all of the following criteria are potentially eligible for study participation: 1. Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing ...
Nomlabofusp is an investigational small molecule being developed for the treatment of Friedreich Ataxia, a rare inherited disease that affects coordination, muscle strength, and other functions. It is administered by subcutaneous injection and is currently in Phase 3 clinical development.
Nomlabofusp targets the underlying cause of Friedreich Ataxia by addressing frataxin deficiency, the molecular defect responsible for the disease. It is designed to restore frataxin levels, which are critically low in patients with this condition.
Nomlabofusp is being developed by Larimar Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol LRMR. The company is focused on advancing this therapy for patients with Friedreich Ataxia.
Nomlabofusp is currently in Phase 3 clinical development. It has received several FDA designations, including Breakthrough Therapy, Accelerated Approval, Orphan Drug, Rare Pediatric Disease, and Fast Track, reflecting its potential to address a serious unmet medical need.
Nomlabofusp is being evaluated in a Phase 3 clinical trial with the identifier NCT07778836. This randomized, double-blind, placebo-controlled study is recruiting approximately 150 participants aged 12 years and older with Friedreich Ataxia in the United States.
Nomlabofusp is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials, meaning it has not yet received marketing authorization. Its FDA designations, including Breakthrough Therapy and Fast Track, are intended to expedite its development and review.