Drugs / Nomlabofusp
Nomlabofusp
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold Friedreich ataxia 2 milestones | |||||
| Clinical hold | US (FDA) | — | Friedreich ataxia | 2024-05-20 | “today announced the U.S. FDA has removed the partial clinical hold previously placed on the company's nomlabofusp (CTI-1601) clinical program.” larimartx.com ↗ |
| Clinical hold | US (FDA) | — | Friedreich ataxia | 2022-09-14 | “in accordance with a partial clinical hold on the CTI-1601 program first put into place after a full clinical hold was lifted in September 2022” larimartx.com ↗ “the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold.” larimartx.com ↗ |
| Clinical hold Indication not stated 2 milestones | |||||
| Clinical hold | US (FDA) | — | — | 2022-02-14 | “FDA stated it is maintaining its clinical hold at this time and that additional data is needed to resolve the clinical hold” larimartx.com ↗ |
| Clinical hold | US (FDA) | — | — | 2021-05-25 | “today announced that the United States Food and Drug Administration (FDA) has placed a clinical hold on the CTI-1601 clinical program” larimartx.com ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT06681766 | Dec 2024 → Apr 2025 | Friedreich ataxia | Larimar Therapeutics, Inc. | Terminated | No outcome recorded Stop: Enrollment |
| Phase 1 | NCT04519567 | Jul 2020 → Mar 2021 | Friedreich ataxia | Larimar Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04176991 | Dec 2019 → Oct 2020 | Friedreich ataxia | Larimar Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT06447025 | Jan 2024 → Jan 2027 expected | Friedreich ataxia | Larimar Therapeutics, Inc. | Recruiting | No outcome recorded |
| Phase 2 | NCT05579691 | Sep 2022 → Dec 2023 | Friedreich ataxia | Larimar Therapeutics, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2026-06-29 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia larimartx.com ↗
One of six non-ambulatory participants at baseline became ambulatory after 1 year of dosing; none of the seven ambulatory participants progressed to non-ambulatory at 1 year |
| 2026-04-30 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Publishes Cross-Species Findings Supporting Skin as a Surrogate for the Measurement of Frataxin in Tissues Clinically Relevant to Friedreich’s Ataxia larimartx.com ↗
Nonclinical data across mice, rats, and non-human primates consistently showed that treatment with nomlabofusp increases frataxin (FXN) levels in tissues clinically relevant to Friedreich’s ataxia (FA). |
| 2026-02-24 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026 larimartx.com ↗
Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s review of available clinical data from open label study |
| 2025-09-29 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia larimartx.com ↗
Consistent directional improvement across 4 key clinical outcomes (mFARS, FARS-ADL, 9-HPT, MFIS) observed after 1 year of nomlabofusp treatment could suggest potential for clinical benefit relative to a worsening in a FACOMS natural history study reference population |
| 2025-07-08 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Publishes Nonclinical Data Supporting the Therapeutic Potential of Nomlabofusp in Patients with Friedreich’s Ataxia larimartx.com ↗
Nonclinical findings provide evidence of the mechanism of action of nomlabofusp and support the potential use of skin FXN concentrations as a novel surrogate endpoint for Larimar’s planned BLA submission in Q2 2026 seeking accelerated approval |
| 2024-12-16 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Announces Positive Initial Data from Ongoing Long-term Open Label Extension Study & Progress Across Nomlabofusp Program for Friedreich’s Ataxia larimartx.com ↗
Daily subcutaneous injections of 25 mg nomlabofusp in 14 participants were generally well tolerated for up to 260 days in the ongoing open label extension (OLE) study |
| 2024-11-18 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Presents Additional Data from Phase 1 Studies and Phase 2 Dose Exploration Study Supporting the Nomlabofusp Clinical Program at ICAR 2024 larimartx.com ↗
Treatment with nomlabofusp modified gene expression and lipid profiles in addition to increasing frataxin (FXN) levels in study participants with Friedreich’s ataxia (FA) |
| 2024-05-30 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Selected by FDA to Participate in START Pilot Program for Nomlabofusp in Friedreich’s Ataxia larimartx.com ↗ |
| 2024-05-20 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Announces FDA has Removed Partial Clinical Hold for Nomlabofusp Program in Friedreich’s Ataxia larimartx.com ↗
The FDA removed the partial clinical hold after a review of data from the Company’s recently completed four-week, placebo-controlled Phase 2 dose exploration study. |
| 2024-02-12 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich’s Ataxia larimartx.com ↗
Nomlabofusp was generally well tolerated following repeated subcutaneous injections in patients in the 25 and 50 mg cohorts with no serious adverse events |
| 2023-08-10 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Reports Second Quarter 2023 Operating and Financial Results larimartx.com ↗
Initiation cleared for 50 mg cohort in Phase 2 Friedreich's ataxia (FA) dose exploration trial following FDA review of unblinded 25 mg cohort Phase 2 data |
| 2023-07-25 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Receives FDA Clearance to Proceed to 50 mg Cohort in CTI-1601’s Phase 2 Friedreich's Ataxia Trial and to Initiate Open Label Extension Trial larimartx.com ↗
In addition, Larimar’s open label extension (OLE) trial was also cleared for initiation by the FDA. |
| 2023-05-15 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Reports Preliminary Top-line Data from Phase 2 Trial’s 25 mg Cohort Showing Increases in Frataxin Levels in Patients with Friedreich’s Ataxia and First Quarter 2023 Financial Results larimartx.com ↗
Data from the cohort indicate CTI-1601 was generally well tolerated and showed increases in frataxin (FXN) levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells) at day 14. |
| 2022-09-14 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Announces FDA Clearance to Initiate the 25 mg Cohort of a Phase 2 Dose Exploration Trial of CTI-1601 in Friedreich’s Ataxia Patients larimartx.com ↗
Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the initiation of the 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. |
| 2022-02-14 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Provides Update on CTI-1601 Clinical Program larimartx.com ↗
FDA stated it is maintaining its clinical hold at this time and that additional data is needed to resolve the clinical hold. |
| 2021-05-25 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Reports FDA Clinical Hold on CTI-1601 and Termination of Recently Announced Private Placement Financing larimartx.com ↗ |
| 2021-05-20 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Receives European Medicines Agency Priority Medicines (PRIME) Designation for CTI-1601 in Friedreich’s Ataxia larimartx.com ↗
Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for Friedreich’s ataxia (FA) and other complex rare diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTI-1601 for the treatment of FA. |
| 2021-05-11 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Reports Positive Topline Phase 1 Clinical Trial Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich’s Ataxia larimartx.com ↗
Data demonstrate proof-of-concept by showing that daily subcutaneous injections of CTI-1601 for up to 13 days resulted in dose-dependent increases in frataxin levels from baseline compared to placebo in all evaluated tissues |
| 2020-12-08 | Larimar Therapeutics, Inc. | Results Larimar Therapeutics Announces Completion of Dosing of the Single Ascending Dose Clinical Trial in Friedreich’s Ataxia Patients and Provides Program Update larimartx.com ↗
Dosing has been completed and based on preliminary data, single subcutaneous injections of CTI-1601 at doses up to 100 mg are thought to have been well tolerated. |
| 2020-07-28 | Larimar Therapeutics, Inc. | Regulatory Larimar Therapeutics Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CTI-1601 for the Treatment of Friedreich’s Ataxia larimartx.com ↗
Larimar Therapeutics, Inc. (Nasdaq:LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for CTI-1601 |
All press releases naming this drug 31 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nomlabofusp | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06681766 ↗ |
| Known as | CTI-1601 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05579691 ↗ |
| Known as | Tat-frataxin | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095325 ↗ |
| Action | Restore | “is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia” NCT04176991 ↗ |
| Modality | Protein / enzyme biologic | “CTI-1601 is a recombinant fusion protein” NCT04176991 ↗ |
| Route | Subcutaneous | “Single Dose/Subcutaneous” NCT04176991 ↗ |