Larimar Therapeutics Completes Rolling BLA Submission Seeking Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia

Larimar plans a Q1 2027 open-label safety update while enrolling patients in its confirmatory trial.

Rhea-AI Summary

Larimar Therapeutics (LRMR) completed its rolling application to the FDA seeking accelerated approval of nomlabofusp for Friedreich’s ataxia. The application relies on increased skin frataxin, a protein deficient in the disease, as a potential surrogate endpoint—a measure intended to predict clinical benefit. FDA is willing to consider this measure; approval remains subject to review.

Previously reported open-label data showed sustained skin frataxin increases, with all evaluable participants at one year reaching levels expected in asymptomatic carriers. Clinical outcomes showed directional improvement relative to worsening in a natural history reference population. Enrollment is underway in the confirmatory FORWARD-FA Phase 3 study. Larimar anticipates submission acceptance in early 2027 and potential U.S. approval in mid-2027, targeting a Q3 2027 launch if approved.

12 points · 0 major

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0 major · 2 points

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointAccelerated approval application completed for nomlabofusp to treat Friedreich’s ataxia.
• Moderate pointSkin frataxin reached asymptomatic carrier ranges in all evaluable participants at one year in the open-label study.
• Moderate point. Forward-looking: it has not happened yet and may not happen.Potential U.S. approval is anticipated by Larimar in mid-2027.
• Minor pointClinical outcomes showed directional improvement at one year relative to worsening in a natural history reference population.
• Minor pointExploratory biological markers showed directional improvement versus placebo in plasma lipids and peripheral tissue gene transcription.

7 minor points

• Minor pointFORWARD-FA enrollment underway in the global confirmatory Phase 3 study to verify clinical benefit.
• Minor point. Forward-looking: it has not happened yet and may not happen.Submission acceptance is anticipated by Larimar in early 2027.
• Minor point. Forward-looking: it has not happened yet and may not happen.Open-label safety update is planned for Q1 2027.
• Minor point. Forward-looking: it has not happened yet and may not happen.U.S. launch is targeted for Q3 2027, if approved.
• Minor point. Forward-looking: it has not happened yet and may not happen.Rare Pediatric Disease Priority Review Voucher acquisition and monetization are potential mid-2027 milestones, if approved.
• Minor pointFDA designations include Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug status.
• Minor pointSTART pilot participation has enabled more frequent FDA interactions supporting development and regulatory strategy.

Negative

• Moderate pointSkin frataxin remains a potential surrogate endpoint; FDA has expressed willingness to consider it, not acceptance.
• Minor pointFDA approval remains subject to review; prior feedback addressed the package’s capacity to support submission and review.

News Explained

Larimar says FDA’s pre-BLA meeting minutes indicated its existing open-label data package appeared capable of supporting submission and review of an accelerated-approval application.

Key Figures

Skin FXN result: All evaluable participants Study duration: 18 months BLA acceptance milestone: Early 2027 +3 more

Skin FXN result

All evaluable participants

At one year, achieved skin FXN levels within the range expected in asymptomatic heterozygous carriers

Study duration

18 months

FORWARD-FA confirmatory Phase 3 study

BLA acceptance milestone

Early 2027

Potential upcoming milestone

Open-label safety update

Q1 2027

Potential upcoming milestone

U.S. approval milestone

Mid-2027

Potential upcoming milestone

U.S. launch target

Q3 2027

Targeting launch if approved

Historical Context

2 past events · Latest: Aug 04

2 events

1. Aug 04

   BLA progress and data

   24h Move

   +3.3%

   FDA feedback supported rolling BLA plans; the update reported sustained FXN increases.

2. Jun 29

   BLA submission and data

   24h Move

   -12.6%

   Larimar filed its first rolling BLA module and reported open-label biomarker and clinical findings.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

biologics license application, accelerated approval, surrogate endpoint, pharmacodynamic markers, +2 more

6 terms

biologics license application regulatory

"completion of its rolling Biologics License Application (BLA) submission"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

accelerated approval regulatory

"seeking accelerated approval of nomlabofusp"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

surrogate endpoint medical

"skin FXN concentrations as a potential novel surrogate endpoint"

A surrogate endpoint is a measurable substitute used in a clinical trial—like a lab test or imaging result—that stands in for a direct patient benefit, such as longer life or improved daily function. Investors care because regulators may accept these quicker, earlier signals to clear or fast-track a treatment, which can shorten development time, reduce costs and change a drug’s market prospects; think of it as using a thermometer to predict recovery instead of waiting for full healing.

pharmacodynamic markers medical

"exploratory pharmacodynamic markers (plasma lipids and peripheral tissue gene transcription)"

Measurements that show a drug or therapeutic intervention is producing a biological effect in the body; these can be molecules, cells, enzyme activities, imaging signals or other lab values that change after dosing and track the drug’s mechanism of action or target engagement. Pharmacodynamic markers indicate that a treatment is doing something biologically expected (and can show dose–response or timing), but they are not by themselves proof of clinical benefit or long‑term safety and often serve as intermediate or surrogate signals used in early clinical trials and regulatory assessments.

double-blind medical

"global confirmatory Phase 3 study, a double-blind, placebo-controlled study"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

subcutaneous medical

"evaluate daily subcutaneous use of nomlabofusp over 18 months"

Subcutaneous means situated or applied just beneath the skin. In finance, the term can describe processes or investments that are hidden or not immediately visible, much like something placed under the skin that isn't easily seen from the outside. Recognizing subcutaneous activities helps investors understand underlying factors that may influence markets or asset values over time.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• Completed BLA submission marks a major milestone in advancing nomlabofusp as the first potential disease-modifying therapy to address the underlying cause of FA
  
  
• Submission follows FDA alignment on key elements of the BLA package for submission, including willingness to consider skin frataxin as a potential novel surrogate endpoint
  
  
• Previously reported open label data showed sustained increases in skin frataxin with directional clinical improvements observed in multiple outcomes relative to a natural history reference population
  
  
• Patients currently enrolling in FORWARD-FA, the global confirmatory Phase 3 study for nomlabofusp

BALA CYNWYD, Pa., Nov. 09, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced completion of its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of nomlabofusp for the treatment of patients with Friedreich’s ataxia (FA). Nomlabofusp is an investigational protein replacement therapy designed to address the underlying cause of FA, frataxin deficiency, by delivering human frataxin to mitochondria.

Completion of the submission follows previously disclosed FDA feedback on key elements of the BLA and represents a significant milestone for Larimar. Larimar is currently enrolling patients in FORWARD-FA, its global confirmatory Phase 3 study, a double-blind, placebo-controlled study that will evaluate daily subcutaneous use of nomlabofusp over 18 months in ambulatory participants ages 2 to 40 who are new to the drug. Approximately two thirds of the participants will be under the age of 21.

“Completing our BLA submission is a major milestone for Larimar and the FA community, bringing years of development work together in an application seeking accelerated approval of nomlabofusp,” said Carole Ben-Maimon, MD, Chief Executive Officer of Larimar. “Our confidence in the program is grounded in the sustained increases in skin frataxin and consistent directional improvements observed in multiple clinical outcomes in patients. It is important to note that patients with FA are expected to experience continued neurologic decline, not improvement. After treatment with nomlabofusp, we have not observed worsening in most participants but rather signs of improvement. Our previously disclosed discussions with FDA have helped inform the content of the submission. As we support the potential FDA review and advance FORWARD-FA, our confirmatory Phase 3 study, our team is preparing for a potential U.S. launch with the goal of making nomlabofusp available to patients as quickly as possible following approval, if granted. In parallel, we continue to evaluate options to optimize our global commercialization strategy.”

The BLA seeks accelerated approval based on increases in skin frataxin (FXN) concentrations as a potential novel surrogate endpoint and is supported by data from the ongoing long-term open label (OL) study. Previously reported findings showed sustained increases in skin FXN, with all evaluable participants at one year achieving skin FXN levels within the range expected in asymptomatic heterozygous carriers. Directional improvements were observed at one year across multiple clinical outcomes relative to worsening in a natural history reference population. Directional improvement of exploratory pharmacodynamic markers (plasma lipids and peripheral tissue gene transcription) compared to placebo treated patients were also observed. Nomlabofusp has the potential to be the first disease-modifying therapy for patients with FA.

“Larimar’s completion of its BLA submission for nomlabofusp marks an important and exciting milestone for the Friedreich’s ataxia community. As the first potential frataxin replacement therapy, nomlabofusp is designed to address the underlying cause of FA. The fact that the submission includes data from adolescents ages 12 to 17 is especially meaningful, as there are currently no approved therapies for children under 16 years of age. FA is a complex and variable disease, and patients and families need multiple treatment options that can address their individual needs across different ages and stages of disease,” said Jen Farmer, M.S., Chief Executive Officer, Friedreich’s Ataxia Research Alliance (FARA).

Submission Highlights:

• Data Package Supporting Submission and Review: FDA reviewed OL study data submitted in a briefing package and confirmed in minutes from the multidisciplinary Type B pre-BLA meeting that the existing data package appears capable of supporting submission and review of a BLA seeking accelerated approval of nomlabofusp as a treatment for patients with FA. Approval will be a matter of review.
• FXN as a Potential Surrogate Endpoint: In those previously disclosed interactions, FDA reaffirmed its willingness to consider FXN as a novel surrogate endpoint and indicated that Larimar’s exposure-response analysis linking nomlabofusp exposure to clinical outcomes is the type of analysis that could support the BLA submission.
• Additional Biological Evidence: FDA previously noted that prospectively collected exploratory gene expression and lipid pharmacodynamic markers data may help further characterize the biological activity of nomlabofusp beyond tissue FXN concentrations.

Potential Upcoming Milestones

• BLA Submission Acceptance: Early 2027
  
  
• Open Label Study Safety Update: Q1 2027
  
  
• U.S. Approval: Mid-2027
  
  
• Obtaining & Monetizing the Rare Pediatric Disease Priority Review Voucher: Mid-2027, if approved
  
  
• U.S. Launch: Targeting Q3 2027 launch, if approved.

Nomlabofusp has received FDA Breakthrough Therapy Designation for the treatment of FA in adults and children as well as Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Nomlabofusp was also selected for FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, which has enabled more frequent interactions with FDA to support our development and regulatory strategy.

About FORWARD-FA
FORWARD-FA, Larimar’s global confirmatory Phase 3 study, is designed to assess safety and clinical outcomes and to verify the clinical benefit of nomlabofusp as part of the Company’s accelerated approval strategy. The double-blind, placebo-controlled study will evaluate daily subcutaneous use of nomlabofusp over 18 months in ambulatory participants ages 2 to 40 who are new to the drug. Approximately two thirds of the participants will be under the age of 21. The focus on younger participants reflects the tendency for more rapid disease progression in this population and underscores the need for evaluating potential treatments for early intervention.

About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.

Forward-Looking Statements

This press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp and any other planned product candidates; the potential of nomlabofusp to address the underlying cause of FA and provide clinical benefit; the interpretation of clinical, biomarker and safety data and their ability to support the BLA; expectations regarding the FDA’s acceptance for filing, review and potential approval of the BLA, including consideration of skin FXN as a potential novel surrogate endpoint; Larimar’s planned research and development efforts, including the design, timing and conduct of FORWARD-FA; interactions with FDA; expectations regarding the potential for, accelerated approval or accelerated access and the impact of such accelerated access on the FDA’s review timeline Larimar’s preparations for and timing of a potential launch, if approved; the potential award and future sale of a Rare Pediatric Priority Review Voucher; and Larimar’s overall development plans, business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations.

In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later non-clinical or clinical trials, and assessments; delays in patient recruitment, including as a result of changes in clinical protocols and adverse events; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy or determine that skin FXN is not an acceptable surrogate endpoint for accelerated approval; that completion of the BLA submission does not assure acceptance for filing or accelerated approval and that FDA may require additional information or studies; Larimar’s ability to realize the benefits of Breakthrough Therapy Designation; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to successfully develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.

Investor Contact: Joyce Allaire      LifeSci Advisors  jallaire@lifesciadvisors.com      (212) 915-2569

Company Contact: Michael Celano  Chief Financial Officer mcelano@larimartx.com (484) 414-2715

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What evidence supports Larimar’s nomlabofusp accelerated approval application?

The application relies on increases in skin frataxin as a potential surrogate endpoint, supported by the ongoing long-term open-label study. All evaluable participants at one year reached levels expected in asymptomatic carriers. Multiple clinical outcomes showed directional improvement relative to worsening in a natural history reference population.

When could Larimar’s nomlabofusp receive FDA approval and launch?

Larimar anticipates potential U.S. approval in mid-2027 and targets a Q3 2027 launch, if approved. Submission acceptance is a potential early 2027 milestone. Completion of the application does not establish FDA approval.

Who is eligible for Larimar’s FORWARD-FA confirmatory trial, and how is it designed?

FORWARD-FA enrolls ambulatory participants ages 2 to 40 who are new to nomlabofusp. The double-blind, placebo-controlled study evaluates daily injections under the skin over 18 months to assess safety and clinical outcomes and verify clinical benefit. Approximately two thirds of participants will be under age 21.

Does Larimar’s nomlabofusp application include adolescent data?

The application includes data from adolescents ages 12 to 17. Inclusion of these data is part of the submitted package, not an approved age indication.