sodium phenylbutyrate / Generic mfg. - LARVOL DELTA

Sodium Phenylbutyrate Ameliorates Ovariectomy-Induced Bone Loss in Rats. (PubMed, Medicina (Kaunas)) - " SPB mitigates OVX-induced oxidative stress, inflammatory cytokine release, and osteoclast-mediated resorption, resulting in partial but significant improvements across biochemical, structural, and histomorphometric parameters in estrogen-deficient rats. Given its established clinical safety profile, SPB emerges as a cost-effective candidate for repurposing in postmenopausal osteoporosis, warranting further translational and clinical studies."

Biomarker • Journal • Preclinical • Osteoporosis • Rheumatology • CTSK • IL6 • TNFA

CRRT in Nonhepatic Hyperammonemic Encephalopathy After Roux-en-Y Gastric Bypass (KIDNEY WEEK 2025) - "After nutrient and vitamin supplementation, L-carnitine, arginine, sodium phenylbutyrate and lactulose, the arterial ammonia normalized within two weeks. Clinicians faced with a similar clinical case can consider dialysis as an initial therapy. Future research should focus on understanding the role of CRRT dose, timing, and modality in management of patients with hyperammonemia."

Bariatric surgery • Cardiovascular • CNS Disorders • Dermatitis • Dermatology • Epilepsy • Hematological Disorders • Hepatology • Hypertension • Immunology

Urea cycle disorders in Argentina, analysis from a cohort of 135 patients diagnosed in the last 20 years. (SSIEM 2023) - "76% required chronic treatment: sodium benzoate 76%, sodium phenylbutyrate 27%, and glycerol phenylbutyrate 12%...The mortality rate was higher than the one reported in other countries. We reported their presenting symptoms and sociocultural characteristics as well as accessibility to diagnosis and treatment, trying to identify the negative correlations in order to portray which variables are most related to worse results."

Clinical • Metabolic Disorders

Sodium Phenylbutyrate as Therapeutics (SSIEM 2023) - No abstract available

Zevra Reports Third Quarter 2025 Financial Results and Corporate Update (The Manila Times) - "MIPLYFFA: Eight prescription enrollment forms during Q3 2025, bringing the total since product launch to 137; OLPRUVA: One prescription enrollment form during Q3 2025, bringing the total since product launch to 30...$26.1 million for Q3 2025, which includes $22.4 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $2.4 million in net reimbursements from the French EAP for arimoclomol..."

Commercial • Genetic Disorders

CircSipa1l1 modulates melanoma cell differentiation by activating the IGF2BP1-ARHGDIB axis and ERK signaling pathway. (PubMed, J Transl Med) - "Our findings reveal that circSipa1l1 acts as an oncogenic circRNA by regulating the IGF2BP1/ARHGDIB/ERK axis in melanoma, suggesting it could be a potential therapeutic target for melanoma differentiation therapy."

Journal • Melanoma • Oncology • Solid Tumor • ARHGDIB • IGF2BP1 • Tyrosinase

Design, Synthesis, Development, and Biological Evaluation of Third-Generation Tubulin Inhibitors for Treatment of Cancer. (PubMed, Curr Protoc) - "The tubulin inhibitors have been synthesized in high yields without incorporating tubuvaline and tubuphenylalanine fragments present in the natural tubulysins, which are known to be essential for their anticancer activity.."

Journal • Oncology

Adverse drug reaction profiles of histone deacetylase inhibitors. (PubMed, Sci Rep) - "This study aimed to explore the global observational adverse drug reaction (ADR) profiles of the HDACIs: vorinostat, belinostat, panobinostat, pracinostat, entinostat, romidepsin, bufexamac and sodium phenylbutyrate. Thrombocytopenia observed with vorinostat, panobinostat and romidepsin may stem from dual inhibition of HDAC1 and HDAC2 and their higher volume of distributions (Vd). Despite all HDACIs having a similar mechanism of action, their unique pharmacology and differing physicochemical and pharmacokinetic properties landscape results in varying ADR profiles."

Adverse drug reaction • Journal • CNS Disorders • Depression • Hematological Disorders • Musculoskeletal Diseases • Psychiatry • Thrombocytopenia • HDAC1 • HDAC2 • HDAC3 • HDAC4 • HDAC9

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (clinicaltrials.gov) - P2 | N=24 | Suspended | Sponsor: Jerry Vockley, MD, PhD | Recruiting ➔ Suspended

Trial suspension • Metabolic Disorders • Pediatrics

Real-World Clinical Experience With Sodium Phenylbutyrate and Taurursodiol at a Single Amyotrophic Lateral Sclerosis Center in the United States. (PubMed, Eur J Neurol) - "Experience in real-world clinical settings can help supplement trial data with information on the drug performance at various stages of disease progression. Adverse events impacted treatment persistence in routine clinical practice, underscoring the need for vigilant monitoring and tailored supportive interventions to optimize treatment adherence."

Journal • Real-world evidence • Retrospective data • Amyotrophic Lateral Sclerosis • CNS Disorders

Perioperative Management of a Patient With Arginase 1 Deficiency Undergoing a Liver Transplant: A Case Report. (PubMed, A A Pract) - "The primary management goals are to (1) reduce the risk for hyperammonemia by administering sodium phenylbutyrate and sodium benzoate to promote nitrogen excretion and (2) prevent catabolism by infusing carbohydrates and lipids. Vigilant monitoring for sodium derangements is necessary because these infusions may lead to hyper- or hyponatremia."

Journal • Cardiovascular • Heart Failure • Metabolic Disorders • Transplantation

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA) (clinicaltrials.gov) - P1 | N=1 | Enrolling by invitation | Sponsor: Jerry Vockley, MD, PhD

New P1 trial

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Jerry Vockley, MD, PhD | Trial completion date: Feb 2027 ➔ Jul 2027

Trial completion date • Metabolic Disorders • Pediatrics

Design, Synthesis, and Cytotoxic Evaluation of New Structurally Simplified and Highly Potent Third-Generation Tubulysin Derivatives. (PubMed, Chemistry) - "These inhibitors are devoid of tubuvaline and tubuphenylalanine fragments previously considered essential for tubulin inhibition activity...The method enables the synthesis of potent tubulin inhibitors by avoiding multistep synthetic and purification procedures, supporting the inhibitor's applicability for large-scale synthesis and potential therapeutic development. The structural modifications at the N-terminal result in the loss of activity from nM to µM range, whereas the C-terminal modification had minimal impact on the potency."

Journal • Cervical Cancer • Oncology • Solid Tumor

NAPVAT: Na-Phenylbutyrate VAscular Trial (clinicaltrials.gov) - P1 | N=30 | Not yet recruiting | Sponsor: University of Pennsylvania | Initiation date: May 2025 ➔ Dec 2025

Trial initiation date • Diabetes • Metabolic Disorders

Repurposing the memory-promoting meclofenoxate hydrochloride as a treatment for Parkinson's disease through integrative multi-omics analysis. (PubMed, NPJ Parkinsons Dis) - "Among these, meclofenoxate hydrochloride (MH) and sodium phenylbutyrate (SP) are indicated to be able to prevent mitochondrial destruction, reduce lipid peroxidation, and protect dopamine synthesis...The interaction of MH with a PD-related protein, sigma1, was confirmed experimentally. Thus, our findings support that MH potentially ameliorates PD by interacting with sigma1."

Journal • CNS Disorders • Movement Disorders • Parkinson's Disease

A clinical efficacy study of Sodium phenylbutyrate in the treatment of diminished ovarian reserve (ChiCTR) - P=N/A | N=20 | Not yet recruiting | Sponsor: Zhongshan Hospital, Fudan University; Zhongshan Hospital, Fudan University

New trial

Cerebral Edema Due to Hyperammonemia: A Case of Suspected Urea Cycle Disorder in Adult Patient (ATS 2025) - "The patient was dialyzed for hyperammonemia, treated with hypertonic saline for cerebral edema, and managed for seizures with levetiracetam, lacosamide, and topiramate...This can then be followed by outpatient amino acid supplementation, dietary protein restriction, in some cases, nitrogen scavenger therapy (such as sodium phenylbutyrate or glycerol phenylbutyrate) and avoidance of medications that raise ammonia levels, such as valproic acid in our patient. Mild cases of UCDs can present in adults during physiologic distress, requiring high suspicion as delays may lead to irreversible cerebral injury or death. In patients with unclear neurologic symptoms, particularly new-onset seizures or cerebral edema checking ammonia levels is crucial for prompt identification and treatment."

Clinical • CNS Disorders • Epilepsy • Metabolic Disorders • Pediatrics • Psychiatry • Schizophrenia

Estimating the Effect of a Discontinued Amyotrophic Lateral Sclerosis Treatment on Time-to-Death Using Sequential Target Trial Emulation (ISPOR 2025) - "OBJECTIVES: A combination product of sodium phenylbutyrate and taurursodiol (SP-T) was FDA-approved for amyotrophic lateral sclerosis (ALS) treatment in September 2022, then later withdrawn in April 2024, following unfavorable Phase 3 trial results... Using the Komodo Research Dataset, a claims database with mortality data, we implemented the target trial framework to emulate a sequence of hypothetical randomized trials comparing SP-T initiators vs non-initiators (standard care [riluzole or edaravone]) in each calendar month from October 2022 to March 2024 to estimate the observational analog of the intention-to-treat effect... We utilized a sequential target trial emulation to improve statistical efficiency in estimating the effect of SP-T initiation on time-to-death. Our results suggest a lower risk for death for ALS patients who initiated SP-T compared with non-initiators."

Amyotrophic Lateral Sclerosis • CNS Disorders

Decoding Clinical Heterogeneity in Ornithine Transcarbamylase Deficiency: Novel Mutations and Therapeutic Outcomes from a Longitudinal Cohort Study (ESPE-ESE 2025) - "Therapeutic trajectories: Medical therapy cohort (n=2): Intermittent hyperammonemia (45-78 μmol/L) persisted in patients receiving sodium phenylbutyrate or arginine/citrulline, correlating with suboptimal adherence (30% missed doses)... This study establishes three critical insights for OTCD management: 1. Diagnostic urgency: Plasma amino acid profiling with urinary orotic acid quantification enables rapid triage of suspected cases. 2."

Clinical • Heterogeneity • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Hepatology • Pediatrics

48-Week Results from the HELIOS Trial: A Phase 2, Open-Label Study Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Wolfram Syndrome (ESPE-ESE 2025) - "HELIOS week 24 results for all 12 participants (Intent-to-Treat) and for the 11 with genetically confirmed WS (Per Protocol) were previously reported. PB&TURSO was generally well-tolerated with diarrhea the most common adverse event. Due to the progressive nature of WS, pancreatic beta cell function, glycemic control, visual function, and overall symptom burden typically worsen over time; however, at Week 24, treatment with PB&TURSO showed overall stabilization or improvement relative to baseline."

Clinical • P2 data • CNS Disorders • Metabolic Disorders • Nephrology • Ophthalmology • Otorhinolaryngology • Type 1 Diabetes Mellitus

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Jerry Vockley, MD, PhD | Initiation date: Apr 2025 ➔ Jul 2025

Trial initiation date • Metabolic Disorders • Pediatrics

Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023. (PubMed, Front Pharmacol) - "Spesolimab, Sodium Phenylbutyrate, Nitisinone and Emapalumab are currently in short supply, and the delivery rate of 16 drugs such as Selumetinib, Sirolimus (tablet), Octreotide, Dimethyl Fumarate and Lanreotide is below 80%...This indicates a generally high level of accessibility to drugs for rare diseases in China. However, attention should be given to improving the supply capacity for drugs that are in short supply and have a low delivery rate."

Journal • Rare Diseases

Evaluation of Additive Neuroprotective Effect of Combination Therapy for Parkinson's Disease Using In Vitro Models. (PubMed, Antioxidants (Basel)) - "We demonstrated that some of the medications, used in combination, can exert an additive neuroprotective effect in preclinical models of PD that is superior to that of each of the compounds individually. This project can lead to the development of the first treatment for PD that can slow or prevent its progression."

Journal • Preclinical • CNS Disorders • Movement Disorders • Parkinson's Disease • NEFH

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Jerry Vockley, MD, PhD | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Metabolic Disorders • Pediatrics