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

BALA CYNWYD, Pa., Nov. 09, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. 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). 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.

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.

Overcoming the challenges of rare disease clinical research

Tunis, S., Caro, J., Abraham, P. et al. Overcoming the challenges of rare disease clinical research. Orphanet J Rare Dis (2026). doi:10.1186/s13023-026-04668-w 

 This review focuses primarily on clinical development and regulatory evidence-generation strategies for rare diseases, while also highlighting how residual uncertainty may affect subsequent health technology assessment, reimbursement, and access decisions. We provide examples of drug approval processes and outline various potential strategies for clinical development of rare disease treatments, highlighting the strengths of each technique and discussing the opportunities to enhance clinical research.

Nationwide Assessment of Friedreich’s Ataxia in Sweden: Epidemiology and Patient-Reported Outcomes

Dobloug S, Jennions E, Svenning P ... Nationwide Assessment of Friedreich’s Ataxia in Sweden: Epidemiology and Patient-Reported Outcomes Parkinsonism & Related Disorders, 152. DOI: 10.1016/j.parkreldis.2026.108448 

 Friedreich’s ataxia (FRDA) is considered the most common hereditary ataxia globally, yet marked variation in prevalence has been reported. FRDA prevalence is considered lower in Northern European countries than in other populations, but contemporary epidemiological data are limited. The aim of this study is to investigate the national prevalence of FRDA in Sweden and to characterize demographic patterns, clinical severity, and quality of life within the Swedish FRDA population.

Guide to Cerebellar Ataxic Disorders

Francisco Javier Arpa Gutiérrez, Francisco Javier Rodríguez de Rivera, Irene Sanz Gallego, Susana Chajma Izquierdo, Guide to Cerebellar Ataxic Disorders, Neurology Perspectives, 2026, 100242, ISSN 2667-0496, doi:10.1016/j.neurop.2026.100242. 

We refer to the main epidemiological data on cerebellar ataxias, preferably focused on Spain, and describe their clinical and paraclinical evaluation. We develop a characteristic pathway: the presentation in the patient of a progressive or stable (non-progressive) cerebellar ataxia, which may have a hereditary origin, be a primary acquired ataxia, or be a secondary ataxia, and we address its diagnosis and management.

DNA double-strand break repair deficiency in Friedreich’s Ataxia: Radioprotective role of Zoledronic Acid and Pravastatin

Rafka Salim Challita, Dana Tohme, Leen Abou Najem, Hussein Khram, Walaa Chebli, Gilbert Antaki, Charbel Feghaly, Hanine Bou Hadir, Rana El-Hassan, Wassim Abou-Kheir, Larry Bodgi, 2199 DNA double-strand break repair deficiency in Friedreich’s Ataxia: Radioprotective role of Zoledronic Acid and Pravastatin, Radiotherapy and Oncology, Volume 218, Supplement 1, 2026, Pages S2619-S2620, ISSN 0167-8140, doi:10.1016/S0167-8140(26)03991-5.

Conference abstract

Adjuvia Therapeutics Initiates Phase 1 Clinical Trial of ATI-105 for Friedreich’s Ataxia

Oct 28, 2026. SAN FRANCISCO--(BUSINESS WIRE)--Adjuvia Therapeutics, a biotechnology company developing novel therapeutics to treat mitochondrial dysfunction and disease, today announced that the first participant has been dosed in its Phase 1 clinical trial of ATI-105, its lead investigational therapeutic candidate for Friedreich’s ataxia (FA). The milestone follows the U.S. Food and Drug Administration's (FDA) acceptance of the company's Investigational New Drug (IND) application in September.Adjuvia plans to advance ATI-105 into Phase 1/2 studies in FA in 2027, with a focus on addressing lethal cardiomyopathy, followed by expansions into Leigh syndrome and primary mitochondrial diseases (PMD) in 2028.

La FDA investiga formalmente el riesgo de cáncer de las dos terapias génicas de Novartis para la atrofia muscular espinal

Published 08 November 2026​. El regulador amplía el escrutinio a Itvisma pese a que no se han notificado casos; tres programas independientes basados en AAV están vinculados a la activación de oncogenes del desarrollo mediante un elemento promotor CAG común. 

La Administración de Alimentos y Medicamentos de Estados Unidos (FDA) anunció formalmente el jueves que investiga un riesgo grave de malignidad asociado a las dos terapias génicas aprobadas de Novartis para la atrofia muscular espinal —Zolgensma e Itvisma— después de que el análisis molecular de tejido tumoral de pacientes tratados con Zolgensma confirmara un mecanismo causal concreto: el ADN del vector viral de la terapia se integra en el genoma cerca de un gen conocido por impulsar el cáncer, denominado GLI1, y lo activa. 

La medida de la FDA, publicada el 6 de noviembre por su Centro de Evaluación e Investigación de Productos Biológicos (CBER), supone la primera ocasión en que un regulador estadounidense caracteriza formalmente una terapia génica comercial basada en AAV como causante de oncogénesis insercional en seres humanos. El riesgo pasa así de ser "teórico" —la formulación que todavía figura en la ficha técnica de Zolgensma— a estar confirmado y sujeto a una investigación activa.

FDA Formally Investigates Cancer Risk From Both Novartis SMA Gene Therapies After GLI1 Oncogene Activation Confirmed in Zolgensma Cases

Published: Nov 07 2026, Regulator extends scrutiny to Itvisma despite zero reported cases; three independent AAV programs now linked to developmental oncogene activation via shared CAG promoter element.

The U.S. Food and Drug Administration formally announced Thursday that it is investigating a serious risk of malignancy linked to both of Novartis's approved gene therapies for spinal muscular atrophy — Zolgensma and Itvisma — after molecular analysis of tumor tissue in Zolgensma patients confirmed a specific causal mechanism: the therapy's viral vector DNA integrating into the genome near a known cancer-driving gene called GLI1 and switching it on. 

 The FDA's action, published November 6 by its Center for Biologics Evaluation and Research (CBER), marks the first time a U.S. regulator has formally characterized a commercial AAV gene therapy as having caused insertional oncogenesis in humans — moving the risk from "theoretical," the language still on Zolgensma's product label, to confirmed and under active investigation.

In vivo frataxin expression of a triplex purified minicircle

Mozafari, N., Milagres, S., Cuellar, R. et al. In vivo frataxin expression of a triplex purified minicircle. Gene Ther (2026). doi:10.1038/s41434-026-00653-x 

 Minicircles (MCs) are non-viral DNA vectors offering improved safety and efficiency over conventional plasmids. We used a novel method to purify MCs using biotin-conjugated triplex-forming and strand-invading oligonucleotides (ONs). A 21 bp H-DNA-forming sequence was incorporated into the plasmids to enhance binding specificity of the ONs. The ON-plasmid complexes were removed using streptavidin magnetic beads. This approach yielded highly purified MCs.

Solid Biosciences Reports Third Quarter 2026 Financial Results and Provides Business Updates

CHARLESTOWN, Mass., Nov. 04, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the third quarter ended September 30, 2026, and provided a business update. 

SGT-212 for Friedreich's ataxia (FA) Five participants have been dosed with SGT-212 in the Phase 1b FALCON clinical trial, with no treatment-related serious adverse events observed as of November 3, 2026. Participant screening and enrollment remain ongoing, and Solid expects to report initial data in the first quarter of 2027, subject to participant enrollment. 

In October, SGT-212 received Orphan designation for the treatment of Friedreich's ataxia from the European Commission, acting upon the positive opinion of the European Medicines Agency's Committee for Orphan Medicinal Products.