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 and CTI-1601. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
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
CTI-1601 FDA Regulatory Events
CTI-1601 is a drug developed by Larimar Therapeutics for the following indication: Friedreich's Ataxia (FA).
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:
- CTI-1601
- Announced Date:
- May 30, 2024
- Target Action Date:
- H2 2025
- Estimated Target Date Range:
- July 1, 2025 - December 31, 2025
- Indication:
- Friedreich's Ataxia (FA)
Announcement
Larimar Therapeutics, announced that BLA submission continues to be targeted for 2H 2025
AI Summary
Larimar Therapeutics has announced that it is still targeting a Biologics License Application (BLA) submission for the second half of 2025. The company’s novel protein therapy, nomlabofusp, is designed to treat Friedreich’s ataxia by delivering frataxin directly to mitochondria, addressing a serious unmet medical need in this rare neurodegenerative disease.
Selected for the FDA’s START pilot program, nomlabofusp benefits from enhanced and more frequent communication with the FDA. This improved interaction aims to accelerate crucial steps in the development process, such as initiating pivotal clinical studies and generating high-quality data for regulatory review. Larimar’s focused strategy underscores its commitment to expediting development and bringing a potentially transformative treatment to patients who face limited options.
Read Announcement- Drug:
- CTI-1601
- Announced Date:
- May 30, 2024
- Indication:
- Friedreich's Ataxia (FA)
Announcement
Larimar Therapeutics, announced that the United States Food and Drug Administration (FDA) has selected nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
AI Summary
Larimar Therapeutics announced that the FDA has selected its novel protein replacement therapy nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. The therapy, which aims to treat Friedreich’s ataxia by delivering frataxin to mitochondria, was chosen based on its potential for clinical benefit in a rare neurodegenerative disease and the readiness of its development program. START, a milestone-driven initiative launched by the FDA, offers enhanced communication between sponsors and the agency, which could accelerate pivotal study initiation and the path toward a Biologics License Application submission. Larimar believes that this advanced collaborative approach will help move nomlabofusp closer to being an effective treatment option, ultimately addressing an unmet medical need for patients suffering from this debilitating condition.
Read Announcement- Drug:
- CTI-1601
- Announced Date:
- May 20, 2024
- Target Action Date:
- Q4 2024
- Estimated Target Date Range:
- October 1, 2024 - December 31, 2024
- Indication:
- Friedreich's Ataxia (FA)
Announcement
Larimar Therapeutics, announced that Interim data from OLE study remains on track for Q4 2024
AI Summary
Larimar Therapeutics is progressing with its ongoing Open Label Extension (OLE) study, which is focused on evaluating nomlabofusp for patients with Friedreich’s Ataxia. In the OLE study, participants initially receive 25 mg of the drug with plans to increase the dose to 50 mg after further analysis of frataxin pharmacodynamics. This approach will help determine if the higher dose can further improve frataxin levels while maintaining safety for patients.
Of particular importance, the company has announced that the interim data from the OLE study remains on track to be released in the fourth quarter of 2024. These forthcoming results will provide valuable insights into the long-term safety and effectiveness of nomlabofusp, guiding future dose adjustments and advancing the treatment development for this rare disease.
Read Announcement- Drug:
- CTI-1601
- Announced Date:
- May 20, 2024
- Target Action Date:
- H2 2025
- Estimated Target Date Range:
- July 1, 2025 - December 31, 2025
- Indication:
- Friedreich's Ataxia (FA)
Announcement
Larimar Therapeutics, announced that Biologics License Application (BLA) submission targeted for 2H 2025
AI Summary
Larimar Therapeutics recently announced plans to submit a Biologics License Application (BLA) for its investigational therapy, nomlabofusp, targeted for the second half of 2025. This submission is a key step in the company’s program to develop a treatment addressing Friedreich’s Ataxia by delivering essential frataxin to patient cells.
The company is also advancing its ongoing open label extension study, which started with daily subcutaneous injections at a 25 mg dose. After further characterization of frataxin pharmacodynamics at this level, Larimar intends to escalate the dose to 50 mg. Interim data from the study is expected by the fourth quarter of 2024. This dose exploration process is designed to better understand the optimal therapy level while ensuring the treatment’s long-term safety and efficacy for patients with this rare disease.
Read Announcement- Drug:
- CTI-1601
- Announced Date:
- May 20, 2024
- Indication:
- Friedreich's Ataxia (FA)
Announcement
Larimar Therapeutics announced the U.S. FDA has removed the partial clinical hold previously placed on the company's nomlabofusp (CTI-1601) clinical program. Nomlabofusp is currently in development for the treatment of patients with Friedreich's Ataxia (FA). Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria.
AI Summary
Larimar Therapeutics announced that the FDA has removed the partial clinical hold on its nomlabofusp (CTI-1601) clinical program. This decision came after a favorable review of Phase 2 study data, which supported the safety of the treatment. Nomlabofusp is a novel protein replacement therapy being developed for patients with Friedreich’s Ataxia (FA). The therapy is designed to address the root cause of FA by delivering the essential protein frataxin directly to the mitochondria, where it is needed to improve cellular function.
The ongoing open label extension (OLE) study initially evaluates a daily subcutaneous injection of 25 mg, with plans to escalate to 50 mg following further characterization of frataxin pharmacodynamics. Interim data from the OLE study is expected by the fourth quarter of 2024, marking an important milestone in the development of this promising therapeutic approach for FA.
Read Announcement
