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baclofen (Fleqsuvy)

✓ Approved

Azurity Pharmaceuticals, Inc. · GABBR1 · Small Molecule

What is baclofen?

baclofen is a small molecule developed by Azurity Pharmaceuticals, Inc.. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesFleqsuvy
CompanyAzurity Pharmaceuticals, Inc.
Drug ClassSmall Molecule
Molecular TargetGABBR1
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Azurity Pharmaceuticals, Inc.

Mechanism of Action

Molecular Targets

baclofen acts on 1 molecular target:

GABBR1gamma-aminobutyric acid type B receptor subunit 1 (GABABR1, GB1)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

baclofen is developed for 3 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Nervous system disordersMultiple sclerosis✓ Approved
Nervous system disordersMuscle spasticity✓ Approved
Injury, poisoning and procedural complicationsSpinal cord injury✓ Approved

Related Research Articles

PubMedTheriogenology2026-10-13

SLC32A1 regulates puberty onset in female mice by modulating GnRH neuron function through Kisspeptin and GABA-GABAB receptor signaling.

Pan Zhihao Z, Zhang Wei W, Han Kejian K, Fu Dongyuan D et al.

This study investigated the role of vesicular γ-aminobutyric acid (GABA) transporter (VGAT/SLC32A1) in initiating puberty in female mice. We first measured the expression of SLC32A1 in the hypothalamic-pituitary-ovary axis of female mice at different developmental stages (infancy, prepuberty, puberty, and adulthood) using real-time quantitative reverse transcription PCR (RT-qPCR) and immunofluorescence. Subsequently, we evaluated the effects of SLC32A1 knockdown on puberty onset in vivo and in vitro, including puberty-related gene expression; proliferation and apoptosis of GT1-7 cells; concentrations of GABA, GnRH, and Kisspeptin; vaginal opening time in female mice; serum concentrations of GABA, progesterone (P4), and estradiol (E2); and ovarian morphology analysis. Finally, we treated Slc32a1 knockdown GT1-7 cells with the GABAB receptor agonist baclofen and assessed changes in puberty-related gene expression and in the concentrations of GnRH, Kisspeptin, and GABA in the culture medium to determine whether SLC32A1 regulates puberty via the GABA-GABAB receptor pathway in vitro. Results revealed that in the hypothalamus of female mice, Slc32a1 mRNA levels declined at puberty, followed by a further decrease in adulthood. In the pituitary, the lowest Slc32a1 mRNA levels were observed during the pubertal and adult stages. In the ovaries, compared with the infant and prepubertal stages, Slc32a1 mRNA levels were significantly reduced in the pubertal and adulthood stages. SLC32A1 was primarily located in the arcuate nucleus (ARC), paraventricular nucleus (PVN) and dorsomedial hypothalamic nucleus (DMH) of the hypothalamus, as well as in the adenohypophysis, the ovarian oocytes and granulosa cells at different developmental stages in female mice. Slc32a1 knockdown in GT1-7 cells increased Gnrh, Gpr54, Igf-1, and Tac2 mRNA expression and cell proliferation, while decreased Sirt1, Gabrb2, and Gabbr1 mRNA levels, and reduced the secretion of GABA, GnRH, and Kisspeptin. Mice with hypothalamic Slc32a1 knockdown exhibited earlier vaginal opening and showed upregulation of Gnrh, Kiss-1, Gpr54, Igf-1, and Tac2 mRNA levels in the hypothalamus, as well as downregulation of Sirt1, Gabrb2, and Gabbr1 mRNA levels; increased Lhβ mRNA in the pituitary; significantly decreased serum GABA levels and significantly elevated serum levels of E2 and P4. In GT1-7 cells, the transcriptional changes in the Gnrh, Gpr54, Igf-1, and Tac2 genes, and Kisspeptin secretion induced by Slc32a1 knockdown were significantly reversed after the addition of baclofen. But it failed to restore the reduced GnRH secretion, it even led to further decreases in GnRH secretion. These findings indicate that SLC32A1 exhibits developmentally dynamic changes along the reproductive axis in female mice, and regulates the function of hypothalamic GnRH neurons; Knockdown of Slc32a1 alters the expression of puberty-related genes in the hypothalamus of female mice, as well as the levels of GABA, E2, and P4 in serum, thereby promoting the onset of puberty. This regulatory effect is partially mediated by the Kisspeptin system through GABAB receptors. This provides novel evidence for the role of SLC32A1 in regulating the onset of puberty.

PMID 42863441
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PubMedJournal of chromatography. A2026-10-09

Cyclization of gabapentinoids as a novel approach for their determination by GC-MS/MS in biological fluids.

Dybowski Michal P MP, Dawidowicz Andrzej L AL, Typek Rafal R, Siwek Krystian K et al.

Gabapentinoids are a group of centrally acting depressant drugs and structural analogues of gamma-aminobutyric acid used to treat neurological disorders. Given their narrow therapeutic window, determining their concentrations in physiological fluids, particularly in human blood plasma, is particularly important for monitoring therapeutic efficacy and ensuring patient safety in routine practice. The results obtained in this study using pregabalin, gabapentin, and baclofen demonstrate that preliminary cyclization is significantly more effective for preparing these gabapentinoids for GC analysis than silylation or methylation. The EDAC/DMTMM mixture was identified as the most suitable catalyst for the cyclization of gabapentinoids. The developed procedure results in complete conversion (near 100%) of the investigated gabapentinoids into their cyclic forms. Importantly, matrix components (i.e. plasma/urine components) do not interfere with the catalyzed cyclization process, and no matrix effect is observed. The proposed method is robust and reproducible (about 98%), and the obtained limits of detection and quantification (lower than 0.9 ng/mL and 3.0 ng/mL, respectively) are comparable to those reported in the literature for standard LC-MS methods and significantly lower than those reported for GC-MS methods involving classical derivatization (silylation or methylation) of these analytes. The applicability of the proposed GC signal-enhancement procedure based on preliminary cyclization was confirmed by the analysis of blood and urine samples containing pregabalin, gabapentin, and baclofen. To the best of our knowledge, this is the first report describing the cyclization of gabapentinoids for their determination in biological fluids by GC; thus, the method represents a novel analytical approach.

PMID 42854462
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PubMedNature neuroscience2026-10-09

Alzheimer's disease target and drug discovery by leveraging multiomics and electronic health data.

Hou Yuan Y, Li Yichen Y, Zhang Pengyue P, Lorincz-Comi Noah N et al.

Despite prolific genomic data from individuals with Alzheimer's disease (AD), translation into treatments has lagged. Here we applied Mendelian randomization to multiple AD genome-wide association studies and functional multiomics datasets to nominate druggable targets, identifying 19 targets for individuals of European ancestry and 7 for African ancestry. This included epoxide hydrolase 2 (EPHX2), for which we identified a protective missense variant (p.Arg287Gln). Experiments in induced pluripotent stem cell-derived neurons and cerebral organoids from patients with AD validated the protective role of p.Arg287Gln via reduced levels of EPHX2 and phosphorylated tau (pTau-181). Pharmacological inhibition of EPHX2 improved cognitive performance in the 5xFAD mouse model of AD. Finally, by leveraging electronic health data from 111,680 patients with mild cognitive impairment, we identified 12 drugs associated with reduced AD incidence, including trazodone (targeting ADRA1A) and baclofen (targeting GABBR1). Thus, integrating multiomics data, functional validation and electronic health data identifies new approaches for treating AD.

PMID 42850312
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PubMedLa Revue de medecine interne2026-10-09

[Hiccups in adults: Pathophysiology, diagnostic approach, and management].

Aubignat Mickael M

Chronic hiccups, defined as lasting more than 48hours, represent an underestimated clinical entity with potentially severe impacts on patients' quality of life. The pathophysiology involves a reflex arc comprising the phrenic and vagus nerves, integrated at the pontomedullary level. Recent advances highlight the role of a central pattern generator and neurotransmitter systems (GABA, dopamine, serotonin) in the genesis and perpetuation of hiccups. Epidemiological data reveal a striking male predominance (82-92% of cases), with an organic cause identified in over 90% of men. Etiologies are dominated by gastrointestinal causes, particularly gastroesophageal reflux disease (up to 80% of cases), followed by central neurological causes including Wallenberg syndrome. The diagnostic approach relies on targeted history and physical examination, complemented by laboratory tests, chest radiography, upper gastrointestinal endoscopy, and brain imaging when clinically indicated. Therapeutic management follows a stepwise approach: etiological treatment, proton pump inhibitors as first-line therapy, followed by baclofen or gabapentin. Refractory cases may require neuroleptics or interventional techniques (phrenic nerve block, acupuncture). Despite the absence of formal guidelines, this review proposes a pragmatic diagnostic and therapeutic algorithm based on current literature evidence.

PMID 42855361
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PubMedFrontiers in pharmacology2026-10-08

GABAB receptor modulation during drug-memory reconsolidation: current evidence and translational priorities for relapse prevention.

Sun Kuisheng K, Deng Li L, Li Tao T, Huang Wenhua W et al.

Relapse after abstinence is sustained in part by drug-associated memories reactivated by cues, contexts, actions, and interoceptive states. Reconsolidation-based interventions seek to modify a retrieved memory during transient lability. GABAB receptors suppress transmitter release, hyperpolarize neurons, and inhibit adenylyl-cyclase-cAMP-protein-kinase-A signaling, providing biological plausibility for temporally aligned modulation. This structured narrative review organizes the literature using a non-comparative evidence-domain framework. Direct retrieval-window evidence remains limited to two local baclofen studies in male mice using morphine conditioned place preference; both reported retrieval dependence, but only the agranular insular cortex study included a delayed-treatment comparison. Earlier systemic baclofen studies conducted during repeated extinction and post-conditioning GABAB positive allosteric modulator studies support memory-related pharmacology but do not isolate a retrieval window. A systemic post-reactivation baclofen study extends the evidence beyond local microinjection, whereas human baclofen and ASP8062 studies define tolerability, exposure, CNS pharmacodynamics, and non-timed clinical boundaries rather than reconsolidation efficacy. Translation requires a validated reactivation procedure, adequate pharmacokinetic coverage and CNS pharmacodynamic engagement, randomized retrieval and timing controls, post-acute persistence, and protection against sedation, task disengagement, and repeated-cue confounding. The evidence supports a falsifiable development hypothesis, not memory erasure or a universal anti-addiction therapy.

PMID 42846061
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PubMedAmerican journal of veterinary research2026-10-07

Intermittent hemodialysis yields the highest baclofen elimination among 5 extracorporeal devices in equine whole blood ex vivo.

Vigh Zsofia Z, Macfarlane Carley-Martin CM, Yeatts James L JL, Baynes Ronald R et al.

To compare baclofen removal by 5 devices across 3 modalities-intermittent hemodialysis (IHD) with low- and high-efficiency dialyzers, prolonged intermittent renal replacement therapy (PIRRT), and 2 hemoperfusion (HP) devices-in ex vivo equine blood. We hypothesized that IHD would be the most effective in baclofen elimination. Heparinized equine whole blood was spiked with reference baclofen and treated with low-efficiency IHD, high-efficiency IHD, PIRRT, carbon HP, polymer HP, and control. Each modality ran for 2 hours, targeting 20 times the assigned blood volume. Plasma baclofen was measured by UPLC-MS-MS at baseline and every 20 minutes. Elimination rate constants (k), reduction ratios, and extraction ratios were calculated from duplicate runs from April 29 through May 2, 2026. Baclofen was eliminated the fastest by IHD (k, almost equal to 2.7 h-1 for both dialyzers; half-life, almost equal to 15 minutes), then PIRRT (k, 2.3 h-1), and most slowly by HP (1.7 and 1.3 h-1 for carbon HP and polymer HP). Between-run variability was lowest for PIRRT (coefficient of variation, 6%) and highest for carbon HP (coefficient of variation, 77%). Reduction ratios were 99.4% (both IHD), 98.9% (PIRRT), 92.7% (polymer HP), and 89.2% (carbon HP). All modalities substantially reduced baclofen in equine blood ex vivo. Based on the elimination rate constant, IHD was the most effective modality for baclofen elimination. Intermittent hemodialysis achieves the most rapid removal. In vivo studies are warranted to compare the performance and efficacy of each modality across clinical settings.

PMID 42843408
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