Larimar Therapeutics' Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Larimar Therapeutics (LRMR).
Over the past two years, Larimar Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
nomlabofusp. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Nomlabofusp FDA Regulatory Timeline and Events
Nomlabofusp is a drug developed by Larimar Therapeutics for the following indication: For Friedreich's Ataxia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- nomlabofusp
- Announced Date:
- February 24, 2026
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp, a frataxin (FXN) protein replacement therapy with disease modifying potential, for the treatment of adults and children with Friedreich's ataxia (FA).
AI Summary
Larimar Therapeutics announced the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to nomlabofusp, a frataxin (FXN) protein‑replacement therapy with potential to modify disease course in Friedreich’s ataxia (FA). The designation covers treatment for both adults and children with FA and reflects early clinical signs that nomlabofusp may offer meaningful improvement over currently available options.
Breakthrough Therapy Designation is meant to speed development and regulatory review for drugs addressing serious conditions when preliminary evidence shows substantial benefit. The FDA’s decision was based on its review of available clinical data from Larimar’s ongoing open‑label study in adult and pediatric FA patients. Larimar plans to continue advancing nomlabofusp through confirmatory trials and regulatory filings toward potential approval and patient access.Read Announcement
- Drug:
- nomlabofusp
- Announced Date:
- September 29, 2025
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics, announced positive 25 mg and 50 mg data from the ongoing long-term open label (OL) study evaluating daily subcutaneous injections of nomlabofusp self-administered or administered by a caregiver in participants with Friedreich's ataxia (FA), a rare, progressive, and systemic disease with neurologic deterioration.
AI Summary
Larimar Therapeutics reported positive data from its ongoing long-term open-label study testing daily subcutaneous injections of nomlabofusp in people with Friedreich’s ataxia (FA), a rare, progressive disease that causes nerve and muscle damage.
Across four completed studies and the open-label trial, 65 participants received at least one dose of nomlabofusp. In the open-label study, 39 people enrolled, 14 have been treated for six months or more, and eight have received daily doses for over a year.
Skin frataxin (FXN) levels rose with both 25 mg and 50 mg doses. All 10 participants with six-month data reached FXN levels above 50% of the median found in healthy volunteers—similar to asymptomatic carriers.
After one year, patients showed consistent directional improvements in four key clinical measures (mFARS, FARS-ADL, nine-hole peg test, and fatigue scale), whereas a natural history study population worsened.
Seven participants had anaphylaxis within six weeks of dosing; most injection site reactions were mild. Following a revised starting dose regimen, long-term nomlabofusp was generally well tolerated. A Biologics License Application is planned for Q2 2026.
Read Announcement- Drug:
- nomlabofusp
- Announced Date:
- September 28, 2025
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics, announced that the Company will host a conference call and webcast to discuss updates for the Company's nomlabofusp clinical development program including data from the ongoing long-term open label study for the treatment of Friedreich's ataxia on Monday, September 29, 2025 at 8:00 am EDT.
AI Summary
Larimar Therapeutics, Inc. plans to host a live conference call and webcast on Monday, September 29, 2025 at 8:00 am EDT. Company leaders will share updates on their nomlabofusp clinical development program, including data from an ongoing long-term open-label study in Friedreich’s ataxia.
Investors and analysts can view the webcast by visiting Larimar’s event link or participate by dialing 1-877-407-9716 (US) or 1-201-493-6779 (international) and using conference ID 13756144. A replay will be available on the Larimar website under “Events & Presentations.”
Larimar Therapeutics is a clinical-stage biotech company focused on developing treatments for complex rare diseases. Nomlabofusp is their lead compound for Friedreich’s ataxia, and the company plans to use its delivery platform to design other therapies for diseases caused by missing or low levels of key bioactive compounds.
Read Announcement- Drug:
- nomlabofusp
- Announced Date:
- July 8, 2025
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics, announced the publication of two peer-reviewed articles highlighting nonclinical data on the therapeutic potential, pharmacology, and mechanism of action of nomlabofusp as a novel frataxin (FXN) protein replacement therapy designed to address the underlying cause of Friedreich's ataxia (FA).
AI Summary
Larimar Therapeutics recently announced the publication of two peer-reviewed articles that provide important nonclinical data on nomlabofusp, a novel frataxin (FXN) protein replacement therapy. The studies highlight nomlabofusp’s mechanism of action and its potential to treat Friedreich’s ataxia by increasing FXN levels in tissues such as the heart, dorsal root ganglia, and skeletal muscle.
The research shows that nomlabofusp can deliver mature and functional frataxin into mitochondria, which is crucial for addressing the underlying cause of the disease. The promising data also supported the FDA’s consideration of using skin FXN concentrations as a surrogate endpoint. Larimar plans to include these insights in their upcoming Biologics License Application submission in Q2 2026, aiming for accelerated approval and providing hope for future treatment options for Friedreich’s ataxia patients.
Read Announcement- Drug:
- nomlabofusp
- Announced Date:
- June 23, 2025
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics announced FDA safety database recommendations and refined timeline for Biologics License Application (BLA) submission to allow for the inclusion of the recommended safety data from adults and children with Friedreich's Ataxia (FA).
AI Summary
Larimar Therapeutics recently announced that the FDA has provided written recommendations for its safety database in support of the nomlabofusp Biologics License Application (BLA). The agency has advised that the safety evaluation should include at least 30 participants with a minimum of 6 months of continuous exposure, and at least 10 of these should have a full year of exposure, with most participants receiving the 50 mg dose. This guidance is designed to ensure that the safety data is robust for both adults and children with Friedreich’s Ataxia (FA).
Based on these FDA recommendations, Larimar has refined its submission timeline and now plans to file the BLA in the second quarter of 2026. This revised plan allows the company to incorporate the recommended safety data from its ongoing clinical trials, including additional data from adolescent participants, into the application for accelerated approval.
Read Announcement- Drug:
- nomlabofusp
- Announced Date:
- December 16, 2024
- Indication:
- For Friedreich's Ataxia
Announcement
Larimar Therapeutics announced positive initial data from the ongoing long-term OLE study evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver in participants with FA.
AI Summary
Larimar Therapeutics recently announced promising initial results from its ongoing open-label extension study for Friedreich’s ataxia. In this study, 14 participants received daily subcutaneous injections of 25 mg nomlabofusp, either self-administered or given by a caregiver, for up to 260 days. The treatment was generally well tolerated, with two serious events that both resolved within 24 hours. Importantly, tissue frataxin levels improved over time—buccal cell levels doubled from a baseline of 15% to 30% and skin cell levels increased from 16% to 72% by Day 90. Early signs also indicate improvements in clinical outcomes, and plasma nomlabofusp levels reached a steady state by Day 30. Encouraged by these results, Larimar is now increasing the dose to 50 mg for some participants in the study.
Read Announcement
Larimar Therapeutics FDA Events - Frequently Asked Questions
As of now, Larimar Therapeutics (LRMR) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Larimar Therapeutics (LRMR) has reported FDA regulatory activity for nomlabofusp.
The most recent FDA-related event for Larimar Therapeutics occurred on February 24, 2026, involving nomlabofusp. The update was categorized as "Designation Grant," with the company reporting: "Larimar Therapeutics, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp, a frataxin (FXN) protein replacement therapy with disease modifying potential, for the treatment of adults and children with Friedreich's ataxia (FA)."
Currently, Larimar Therapeutics has one therapy (nomlabofusp) targeting the following condition: For Friedreich's Ataxia.
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FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:LRMR last updated on 2/24/2026 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
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