Sentinel (cenegermin-bkbj) / Dompe - LARVOL DELTA

Real-world landscape of drug-related ocular injuries: a retrospective pharmacovigilance study. (PubMed, Front Pharmacol) - "The top three drugs by signal strength were brolucizumab (ROR = 132.15), macrogol 400 (ROR = 117.96), and cenegermin (ROR = 60.19)...TTO analysis indicated that most drugs exhibited an "early failure type" (β < 1), such as dupilumab (β = 0.68); only two drugs exhibited a random failure type. The risk of drug-related ocular injuries is primarily associated with sensory organ drugs and biologics, with the greatest risk occurring during the early treatment phase. Clinical monitoring should prioritize female and elderly patients, especially regarding ocular symptoms at the onset of drug therapy, to strengthen pharmacovigilance and inform personalized medication."

Adverse events • Journal • Real-world evidence • Retrospective data • Cataract • Oncology • Ophthalmology

Current State of the Neurotrophin-Based Pharmaceutics in the Treatment of Neurodegenerative Diseases and Neuroinflammation. (PubMed, Med Sci (Basel)) - "The study identified limitations and future prospects for neurotrophin-based pharmaceuticals. For future clinical trials, attention should be paid to the pharmacogenetic profiles of the patients and the evaluation of the inflammatory status of the disease. Novel plasma biomarkers of the effectiveness are needed as well as TSPO-PET imaging. Drug delivery systems remain insufficient; therefore, efforts should focus on inducing endogenous neurotrophin production and developing highly selective agonists and antagonists of neurotrophin receptors. It is crucial to establish a favorable premorbid background before neurotrophin therapy to minimize immunoresistance."

Journal • Review • Alzheimer's Disease • CNS Disorders • Gene Therapies • Inflammation • Mental Retardation • Movement Disorders • Neuralgia • Ophthalmology • Pain • Parkinson's Disease • Psychiatry

Long-term outcomes of corneal neurotization for neurotrophic keratopathy: a dual center, single surgeon retrospective study. (PubMed, Plast Reconstr Surg) - "Corneal neurotization is highly effective and well-tolerated for managing NK, promoting a robust ocular surface that facilitates future rehabilitative surgeries."

Journal • Retrospective data • Dry Eye Disease • Ophthalmology

Targeted Therapies and Pharmacologic Advances in Mucous Membrane Pemphigoid: A Comprehensive Review. (PubMed, Dermatol Pract Concept) - "Considering the severity of the condition, progressive fibrosis, and resistance to therapy, more research is required in relation to the pathogenesis of MMP and the efficacy and safety profile of novel pharmacological options. Pharmacological agents should provide the achievement and maintenance of remission with minimal adverse effects. A broader spectrum of pharmacological agents will allow a personalized approach and more alternatives, in particular for recalcitrant cases, failure of the previous therapy, and in patients with MMP and malignancy."

Journal • Review • Dermatology • Fibrosis • Immunology • Infectious Disease • Oncology

Outcome of Cenegermin in the Treatment of Neurotrophic Keratopathy. (PubMed, Cornea) - "Cenegermin is an effective treatment of NK in the clinical setting, although a notable proportion of patients experience disease recurrence subsequently. Mackie staging is a significant prognostic factor for treatment success."

Journal • Dry Eye Disease • Ophthalmology

Dompé plans to apply the voucher to its Biologics License Application (BLA) for an intranasal form of Nerve Growth Factor (Cenegermin-bkbj) for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION). (Businesswire) - "With this selection, Dompé receives a voucher shortening the marketing application review from the standard 10–12 months to just 1–2 months."

FDA event • Ophthalmology

Evaluation of in vitro and in vivo release of recombinant human nerve growth factor from bioengineered human stromal lenticule. (PubMed, Front Bioeng Biotechnol) - "Bioengineered lenticules exhibit sustained rhNGF release for at least 7 days in vitro and up to 1 month in vivo. These results, together with absence of adverse effects, and significant increase in CNFD at 4 weeks after lenticule implantation suggest its promising potential for clinical use."

Journal • Preclinical

Cenegermin for the Treatment of Moderate or Severe Neurotrophic Keratopathy: Results from a Prospective, Phase IV, Open-Label Study in China. (PubMed, Ophthalmol Ther) - "These findings demonstrate that cenegermin eye drops are an effective, generally well-tolerated treatment in Chinese patients with stage 2/3 NK, supporting their use in this patient population."

Clinical • Journal • P4 data • Dry Eye Disease • Keratitis • Ocular Inflammation • Ophthalmology • Pain

Corneal Neurotization vs. Cenergermin for Neurotrophic Keratitis: A Pilot Study (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

New trial • Keratitis • Ocular Inflammation • Ophthalmology

Topical Cenegermin for Pediatric Neurotrophic Keratopathy: A Review of the Literature and Systematic Review. (PubMed, Cornea) - "Topical cenegermin seems to be an effective option for treating pNK. However, the current literature is limited, and prospective controlled studies are needed to study its effects further."

Journal • Dry Eye Disease • Keratitis • Ocular Inflammation • Ophthalmology • Pediatrics • Transplantation

Cenegermin treatment for pediatric neurotrophic keratopathy: a systematic review. (PubMed, Can J Ophthalmol) - "Cenegermin is a viable treatment option for pediatric NK, showing efficacy in improving corneal transparency and, to a lesser extent, visual acuity. However, its adoption is constrained by limited pediatric data, cost, and potential side effects. Further research is needed to establish long-term safety and effectiveness, and to optimize treatment protocols for this vulnerable population."

Journal • Corneal Abrasion • Dry Eye Disease • Infectious Disease • Ophthalmology • Pediatrics

Utility of cenegermin for the management of neurotrophic keratopathy after penetrating keratoplasty. (PubMed, Oman J Ophthalmol) - "Following an 8-week course of cenegermin, patients with a history of PK demonstrated improvement in NK stage, epithelial healing, and reduced epithelial defect size lasting 6 months after treatment completion. Patients with prior PK and NK may be considered candidates for cenegermin treatment."

Journal • Dry Eye Disease • Ophthalmology • Transplantation

The Role of Nerve Growth Factor on the Ocular Surface: A Review of the Current Experimental Research and Clinical Practices. (PubMed, Int J Mol Sci) - "Notably, recombinant human NGF (rhNGF, cenegermin) has been approved as the first topical biologic therapy for NK. Despite encouraging clinical outcomes, challenges such as high treatment costs, limited long-term data, and potential proangiogenic effects remain. This review consolidates current evidence on the role of NGF in ocular surface health and disease, highlighting its biological mechanisms, clinical applications, and future therapeutic potential."

Journal • Review • Cataract • Conjunctivitis • Dry Eye Disease • Keratitis • Ocular Infections • Ocular Inflammation • Ophthalmology • Transplantation

Selective Modulation of Trk Receptors by Cyclo-Organopeptides. (PubMed, ACS Chem Neurosci) - "Only humanized recombinant NGF has been clinically approved: Cenegermin for treatment of neurotrophic keratitis (NK) in the eye...This paper describes how those new cyclo-organopeptides were designed, prepared, and then selected via screens on Trk-transfected cells. It also outlines and explains obstacles which limit progress in this type of study."

Journal • Dry Eye Disease • Keratitis • Ocular Inflammation • Ophthalmology • BDNF

Real-World Safety Profile of Cenegermin Per FDA Adverse Event Reporting System. (PubMed, J Ocul Pharmacol Ther) - "Involvement of other organ systems is highly unlikely. Patients using topical cengermin should be counseled accordingly."

Adverse events • Journal • Real-world evidence • Keratitis • Ocular Inflammation • Ophthalmology • Pain

DOMPE -MT Neurotrophic Keratitis (clinicaltrials.gov) - P4 | N=10 | Active, not recruiting | Sponsor: Toyos Clinic

New P4 trial • Dry Eye Disease • Keratitis • Ocular Inflammation • Ophthalmology

Corneal Nerves After Treatment With Cenegermin (clinicaltrials.gov) - P=N/A | N=8 | Completed | Sponsor: University of California, Los Angeles | Active, not recruiting ➔ Completed | N=40 ➔ 8

Enrollment change • Trial completion • Keratitis • Ocular Inflammation • Ophthalmology

1953-2023. Seventy Years of the Nerve Growth Factor: A Potential Novel Treatment in Neurological Diseases? (PubMed, Aging Dis) - "In these instances, NGF holds promise for promoting tissue regeneration and repair. Challenges persist in addressing the complexity of neurodegenerative pathologies for a combined therapeutic approach."

Journal • Review • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • Cardiovascular • CNS Disorders • Gene Therapies • Huntington's Disease • Movement Disorders • Musculoskeletal Diseases • Oncology • Orthopedics • Parkinson's Disease • Sarcoma • Solid Tumor • Vascular Neurology

Long-Term Therapeutic Effects of Topical Recombinant Human Nerve Growth Factor in Stage I Neurotrophic Keratitis and Retreatment Considerations (ARVO 2025) - "Purpose To evaluate the long-term therapeutic effects of topical recombinant human nerve growth factor (rhNGF; cenegermin) in patients with stage I neurotrophic keratitis (NK)...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Keratitis • Ocular Inflammation • Ophthalmology

Trends and Insights of a Tertiary Referral Hospital for Patients with Neurotrophic Keratopathy (ARVO 2025) - "Purpose To examine the impact of the FDA approval in 2018 and subsequent use of Cenegermin 0.002% since 2019 on referral patterns for patients with neurotrophic keratopathy (NK) by comparing the initial clinical findings observed at a tertiary referral hospital...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Dry Eye Disease • Keratitis • Ocular Inflammation • Ophthalmology

Topical application of the HSV-1-based gene therapy vector KB801 results in sustained nerve growth factor expression in wounded murine corneas (ARVO 2025) - "The only approved therapeutic option for NK is cenegermin-bkbj, an ophthalmic solution of recombinant human NGF (rhNGF) administered six times daily for eight weeks; however, the high dosing frequency that is required due to rapid rhNGF clearance is burdensome and may lead to suboptimal treatment outcomes...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • Preclinical • Keratitis • Ocular Inflammation • Ophthalmology • NGF

Topical Cenegermin-Bkbj Dramatically Resolves Neurotrophic Keratitis Associated With Pars Plana Vitrectomy. (PubMed, Ophthalmic Surg Lasers Imaging Retina) - "A 60-year-old woman with recurrent retinal detachments in the right eye after receiving the third PPV developed persistent irritation, photophobia, and worsening visual acuity due to iatrogenic NK despite lubrication, bandage contact lens, preservative-free artificial tears, restasis, and right lower lid punctal cautery over 6 weeks. This case highlights the importance of early recognition, tailored management, and the potential efficacy of Cenegermin-Bkbj in treating NK arising after retinal procedures. [Ophthalmic Surg Lasers Imaging Retina 2025;56:XX-XX.]."

Journal • Keratitis • Ocular Inflammation • Ophthalmology • Retinal Disorders

CORNEAL NEURTIZATION: A SAFE AND EFFECTIVE LONG-TERM OPTION FOR NEUROTROPHIC KERATOPATHY. (WRMC 2025) - "The study also aims to determine if use of the human recombinant nerve growth factor therapy cenegermin improves recovery in CN patients...Corneal neurotization yields more favorable outcomes in patients with a peripheral etiology of NK than patients with a central etiology. Patients undergoing corneal neurotization can experience significant improvement in corneal sensation and integrity, allowing them to taper medical therapy and obtain visually rehabilitative procedures if necessary."

Dry Eye Disease • Ophthalmology

Drug Delivery in PROSE Device as Alternative to Frequent Drop Administration in Severe Ocular Surface Disease. (PubMed, Eye Contact Lens) - "The design of the prosthetic replacement of the ocular surface ecosystem (PROSE) device allows it to serve as a novel drug delivery system. In this article, we describe the off-label administration of amphotericin B and cenegermin by instillation in the PROSE device reservoir for the treatment of Candida keratitis in the setting of a persistent epithelial defect."

Journal • Keratitis • Ocular Inflammation • Ophthalmology

REDUCO: A Study to Evaluate Safety and Efficacy of a New Formulation of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drop Solution in Patients With Dry Eye Disease (clinicaltrials.gov) - P2 | N=317 | Completed | Sponsor: Dompé Farmaceutici S.p.A | Active, not recruiting ➔ Completed

Trial completion • Dry Eye Disease • Ophthalmology